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Systemic scleroderma

Systemic scleroderma, also known as systemic sclerosis, is a rare autoimmune disease characterized by excessive collagen production leading to fibrosis, which causes the hardening and tightening of the skin and connective tissues, and often involves internal organs such as the lungs, heart, esophagus, and kidneys. This condition arises from a complex interplay of immune system dysfunction, vascular abnormalities, and tissue fibrosis, resulting in progressive damage that can significantly impair quality of life. It affects approximately 50 to 300 people per million worldwide, with a higher prevalence in women (female-to-male ratio of about 4:1 to 5:1) and peak onset between ages 30 and 50. The disease is classified into two main subtypes based on the extent of skin involvement: limited cutaneous systemic sclerosis (often associated with —calcinosis, Raynaud's phenomenon, esophageal dysmotility, , and ), which typically affects the skin of the face, hands, and forearms with slower progression, and diffuse cutaneous systemic sclerosis, which involves widespread skin thickening and more rapid onset of organ complications. A less common variant, systemic sclerosis sine scleroderma, features internal organ involvement without significant skin changes. Systemic scleroderma differs from localized scleroderma (), which is confined to the skin and does not affect internal organs. Common symptoms include Raynaud's phenomenon, affecting over 95% of patients, where fingers and toes turn white, blue, or red in response to cold or stress due to vasospasm; skin thickening starting in the fingers (sclerodactyly) and spreading proximally; and gastrointestinal issues like heartburn, difficulty swallowing, and bloating from esophageal dysmotility. Other manifestations involve joint pain, fatigue, digital ulcers, telangiectasias (dilated small blood vessels), and calcinosis (calcium deposits under the skin). Complications can be severe, including interstitial lung disease, pulmonary arterial hypertension, scleroderma renal crisis (leading to kidney failure), cardiac arrhythmias, and increased risk of malignancy. The exact cause remains unknown, but it involves —such as variations in genes like HLA, IRF5 (linked to diffuse type), and STAT4 (linked to limited type)—combined with environmental triggers like exposure to silica dust, solvents, or certain viruses (e.g., ). Risk factors include female sex, age 30–50, and certain ethnic backgrounds, with individuals experiencing earlier onset and more severe disease. Although there is no cure, early and management of symptoms and organ involvement are crucial for improving outcomes.

Classification

Subtypes

Systemic scleroderma, also known as systemic sclerosis (), is primarily classified into subtypes based on the extent of involvement and patterns of organ manifestations, which help predict course and guide management. Limited cutaneous systemic sclerosis (lcSSc) is characterized by thickening confined to areas distal to the elbows and knees, including the face and hands, while sparing the trunk and proximal limbs. This subtype often includes features of the , an acronym for (calcium deposits in the ), Raynaud's phenomenon ( causing color changes in fingers and toes), esophageal dysmotility (impaired and ), sclerodactyly (tightening of on fingers), and (dilated small blood vessels on the ). lcSSc accounts for approximately 60-70% of SSc cases and is associated with a slower progression compared to other forms. Diffuse cutaneous systemic sclerosis (dcSSc) involves more extensive skin fibrosis, affecting the trunk, proximal extremities (above elbows and knees), and face, leading to greater functional impairment. Patients with dcSSc face a higher risk of early and severe internal organ involvement, such as rapid progression to or renal crisis. This subtype comprises about 25-40% of cases and follows a more aggressive course with increased mortality risk. Systemic sclerosis sine scleroderma (ssSSc) represents a variant without significant skin thickening, yet it features the hallmark internal organ , vascular abnormalities like Raynaud's phenomenon, and serological markers typical of . This form is less common, affecting fewer than 10% of patients, and may be underdiagnosed due to the absence of cutaneous signs. Overlap syndromes occur when coexists with other connective tissue diseases, such as (leading to inflammatory joint involvement) or (muscle inflammation), and include entities like characterized by overlapping features and high titers of anti-U1 RNP antibodies. These syndromes account for around 10% of cases and complicate classification due to mixed clinical presentations.

Autoantibodies and their implications

Systemic scleroderma, also known as systemic sclerosis (), is characterized by the presence of specific autoantibodies that play a crucial role in serological classification and prognostic assessment. These autoantibodies are detected in over 90% of patients and are generally mutually exclusive, with co-occurrence being rare in more than 90% of cases. The major SSc-specific autoantibodies include (ACA), anti-topoisomerase I antibodies (ATA, also known as anti-Scl-70), and anti-RNA polymerase III antibodies (anti-RNAP3). ACA are found in 50-90% of patients with cutaneous SSc (lcSSc) and are strongly associated with pulmonary arterial hypertension (PAH), occurring in 10-20% of ACA-positive cases, as well as . ATA are present in 30-60% of patients with diffuse cutaneous SSc (dcSSc) and are linked to (ILD), digital ulcers, and an increased of scleroderma renal crisis (SRC). Anti-RNAP3 antibodies occur in 20-25% of dcSSc patients and confer a high of SRC (up to 50%), rapid skin progression, (GAVE), and malignancy within two years of disease onset in approximately 9% of cases. Other relevant autoantibodies include anti-U3 ribonucleoprotein (anti-U3 RNP, or anti-fibrillarin), anti-Th/To, and anti-PM/Scl. Anti-U3 RNP antibodies are detected in 4-10% of SSc patients, predominantly in dcSSc, and are associated with severe disease, including PAH, ILD, and gastrointestinal involvement, particularly in Afro-Caribbean males. Anti-Th/To antibodies appear in 4-13% of ACA-negative lcSSc patients and correlate with ILD and PAH. Anti-PM/Scl antibodies, found in about 4% of cases, are typically associated with overlap syndromes featuring , ILD, and , often with a relatively favorable for involvement. These autoantibodies have significant implications for risk stratification and clinical management. For instance, ATA positivity predicts worse outcomes in skin fibrosis and involvement, guiding intensified monitoring for ILD, while anti-RNAP3 status prompts early screening for and cancer. Prevalence varies by ethnicity; is more frequent and associated with severe disease in compared to other groups, whereas ACA is more common in Caucasians and anti-U3 RNP in Afro-Caribbean populations. Detection of these autoantibodies relies on methods such as indirect immunofluorescence (IIF) on HEp-2 cells for initial (ANA) screening, enzyme-linked immunosorbent assay () for specific quantification (e.g., anti-RNAP3 and ), and line blot assays for multiplex detection. serves as the gold standard for confirming specificity. Testing for SSc-related autoantibodies—specifically ACA, , and anti-RNAP3—is recommended at as part of the 2013 ACR/EULAR criteria, where their presence contributes 3 points toward a total score of ≥9 for definite SSc .

Signs and symptoms

Cutaneous and vascular manifestations

Cutaneous manifestations in systemic scleroderma typically begin with an early edematous phase characterized by non-pitting swelling of the fingers and hands, often accompanied by intense pruritus that can significantly impair quality of life. This initial phase progresses to fibrosis, resulting in progressive skin thickening and tightening, particularly affecting the distal extremities. The extent of skin involvement is quantified using the modified Rodnan skin score (mRSS), a validated semiquantitative measure assessing skin thickness at 17 body sites on a scale from 0 (normal) to 3 (severe), yielding a total score ranging from 0 to 51; higher scores correlate with more extensive fibrosis. Skin tightening often manifests as sclerodactyly, involving distal to the metacarpophalangeal joints of the fingers, which may extend proximally in diffuse cutaneous systemic sclerosis (dcSSc) to include the forearms, upper arms, , and legs, distinguishing it from the more distal involvement in cutaneous systemic sclerosis (lcSSc). Additional cutaneous features include with a characteristic salt-and-pepper appearance due to areas of amid hyperpigmented patches, particularly on the and . In later stages, the skin may soften with , leading to ulceration at sites of , such as over joints. , involving subcutaneous calcium deposits that can cause pain and ulceration, occurs in 18-49% of cases and is more prevalent in lcSSc. Vascular manifestations are prominent and often precede other symptoms, with Raynaud's phenomenon affecting over 95% of patients and serving as an initial hallmark. This vasospastic disorder features triphasic color changes in the digits—pallor due to ischemia, followed by from deoxygenation, and rubor upon reperfusion—typically triggered by cold exposure or emotional stress. Chronic vascular damage contributes to digital ulcers in 20-60% of patients, which are painful ischemic lesions primarily on the fingertips, and may heal with pitting scars. Telangiectasias, dilated superficial capillaries appearing as red spots on the skin of the face, hands, and mucous membranes, are common and more frequent in patients with (ACA). Nailfold capillaroscopy reveals characteristic microvascular abnormalities, including giant capillaries (dilated loops >50 micrometers), hemorrhages, and avascular areas indicating capillary dropout, which reflect ongoing vasculopathy and correlate with digital ulcers. Pruritus in the early phase remains a persistent challenge, exacerbating discomfort during skin remodeling.

Musculoskeletal involvement

Musculoskeletal involvement is a common feature of systemic scleroderma (SSc), affecting 40-90% of patients and serving as a major contributor to functional disability, particularly in hand mobility and daily activities. This involvement encompasses abnormalities in joints, muscles, and bones, often manifesting early in the disease course and progressing to irreversible changes that impair quality of life. Joint symptoms are prevalent, with arthralgias reported in 60-80% of patients, often accompanied by non-erosive and flexion contractures that limit . friction rubs, a characteristic finding in diffuse cutaneous SSc (dcSSc), occur in 20-65% of cases and are associated with skin thickening and reduced joint function. Early in SSc frequently mimics , presenting as symmetric , and is more common in overlap syndromes such as scleromyositis. These joint changes profoundly impact hand function, leading to decreased and dexterity. Muscle involvement affects 10-20% of patients with SSc, typically manifesting as proximal and inflammatory , which may overlap with conditions like and is linked to autoantibodies such as anti-PM/Scl. can result from disuse secondary to joint contractures or direct fibrotic changes, further exacerbating weakness and fatigue. Bone alterations include acro-osteolysis, characterized by resorption of the distal phalanges, observed in approximately 20% of patients and particularly associated with digital ulcers and severe Raynaud's phenomenon. Radiographic findings often show joint space narrowing without erosions, reflecting rather than destructive . These skeletal changes contribute significantly to hand and overall in SSc.

Gastrointestinal tract

Gastrointestinal tract involvement is one of the most common manifestations of systemic scleroderma (), affecting up to 90% of patients and contributing significantly to morbidity through motility disorders and nutritional deficits. Esophageal dysfunction is particularly prevalent, occurring in 70-90% of cases, and is more frequent in limited cutaneous (lcSSc), including the variant where esophageal dysmotility is a defining feature. Esophageal involvement typically results from atrophy and , leading to aperistalsis and impaired lower esophageal function. This dysmotility manifests as , , and severe (), which can progress to erosive , , or peptic strictures in advanced cases. Esophageal manometry often reveals absent peristalsis in the distal two-thirds of the esophagus, with reduced or absent contractility noted in over 50% of affected patients. A major complication is , arising from chronic reflux and impaired clearance, which increases the risk of respiratory infections. Gastric and small bowel complications affect 40-70% of SSc patients, primarily through hypomotility and stasis. , characterized by delayed gastric emptying, occurs in approximately 47-70% of individuals and leads to symptoms such as , early , and . In the , reduced promotes bacterial overgrowth in 30-62.5% of cases, resulting in , , and unintended weight loss. Pseudo-obstruction, a severe form of intestinal hypomotility mimicking mechanical blockage, develops in approximately 4% of patients, more common in diffuse cutaneous systemic sclerosis. Large bowel involvement is less dominant but impacts 20-50% of SSc patients, often presenting with colonic and wide-mouth diverticula. These diverticula, observed in about 42% of cases, arise from and increase the risk of or . Common symptoms include severe due to delayed transit, affecting 20-50% of individuals, alongside in 20-70%, which stems from weakened tone. Overall, these gastrointestinal changes contribute to chronic and reduced in SSc.

Pulmonary and cardiac systems

Pulmonary involvement is a leading cause of morbidity and mortality in systemic scleroderma (), manifesting primarily as (ILD) and pulmonary arterial (PAH). ILD affects 40-80% of patients, with higher prevalence in diffuse cutaneous SSc (dcSSc) at approximately 53%, compared to 35% in limited cutaneous SSc. Common symptoms include progressive dyspnea on exertion and non-productive cough, often developing insidiously in the early disease course. The predominant histological and radiographic pattern is (NSIP), observed in up to 76% of biopsied cases. Anti-topoisomerase I antibodies () are strongly associated with ILD development and progression, particularly in dcSSc patients. Screening for ILD typically involves serial pulmonary function tests (PFTs) to assess restrictive patterns and reduced (DLCO), alongside (HRCT) for early detection of ground-glass opacities and reticular abnormalities. PAH occurs in 8-12% of SSc patients, confirmed by right heart catheterization, and carries a poor due to progressive right ventricular strain. Symptoms overlap with ILD but emphasize exertional dyspnea and , often without prominent . Anti-centromere antibodies (ACA) confer increased risk for PAH, contrasting with the ILD association of ATA. PAH can lead to cor pulmonale, characterized by right heart from chronic pulmonary vascular resistance, with right ventricular dysfunction evident in up to 38% of affected individuals. Cardiac involvement in SSc primarily affects the myocardium through patchy and microvascular ischemia, occurring in 15-35% of patients and independently worsening survival. This disrupts conduction pathways, resulting in arrhythmias such as ventricular tachyarrhythmias in about 15% of cases, and bradyarrhythmias requiring pacing. Systolic dysfunction is less common at around 5-6%, while diastolic dysfunction predominates at 30-35%, contributing to heart failure symptoms like and . , syncope, and exertional intolerance are key clinical signs, often detected via or . remains rare, with effusions noted in only 5-16% without hemodynamic compromise.

Renal and other organs

Scleroderma renal crisis (SRC) affects approximately 10 to 15 percent of patients with diffuse cutaneous systemic sclerosis (dcSSc), occurring less frequently in limited cutaneous systemic sclerosis (lcSSc). This condition is particularly prevalent in patients with anti-RNA polymerase III antibodies, where the risk is substantially elevated compared to those without these autoantibodies. SRC typically involves a with malignant , often accompanied by featuring endothelial damage, platelet-fibrin thrombi in renal arterioles, and in nearly half of cases. A rare normotensive variant of SRC exists but occurs infrequently, without the hallmark severe . Onset of SRC generally occurs within the first 4 to 5 years after systemic sclerosis , underscoring the need for vigilant through regular home measurements and serum assessments to facilitate early detection. Hepatic involvement in systemic sclerosis is uncommon and typically mild, with primary biliary cholangitis (PBC) overlap syndrome observed in 10 to 20 percent of patients with limited cutaneous systemic sclerosis (lcSSc), often linked to anticentromere antibodies. This overlap presents with cholestatic liver enzyme elevations and antimitochondrial antibodies, distinguishing it from direct fibrotic changes in systemic sclerosis. Nodular regenerative hyperplasia, a vascular liver lesion driven by microvascular alterations, represents another rare manifestation, potentially leading to non-cirrhotic portal hypertension without significant fibrosis. Unlike the biliary destruction in PBC overlap, hepatic fibrosis in systemic sclerosis alone is generally less pronounced, with moderate fibrosis detectable in only a subset of cases on biopsy. Neurological involvement in systemic sclerosis primarily affects the peripheral nervous system, with trigeminal neuropathy being the most common cranial manifestation, causing in the trigeminal distribution due to ischemic or fibrotic nerve compression. Carpal tunnel syndrome frequently arises from perineural fibrosis and synovial thickening, contributing to entrapment and hand symptoms. Central nervous system vasculitis is rare, occasionally presenting with headache, seizures, or , but it lacks the widespread prevalence seen in other . Other organ systems can exhibit involvement, including sicca syndrome in 60 to 80 percent of patients, characterized by dry eyes and mouth from glandular akin to Sjögren's syndrome features. autoimmunity is also common, with autoimmune disease prevalent in up to 25 percent of systemic sclerosis cases, often manifesting as and overlapping with sicca symptoms.

Pathogenesis

Etiology and risk factors

The etiology of systemic scleroderma, also known as systemic sclerosis (), remains incompletely understood, with no single causative agent identified. Instead, it is considered a arising from complex interactions between , environmental triggers, and immune dysregulation. Genetic factors contribute significantly to susceptibility, with associations observed in the (HLA) complex. For instance, the HLA-DRB1*1104 is linked to increased risk, conferring an of approximately 2.8 overall and up to 4-fold in anti-topoisomerase I positive cases compared to non-carriers. Familial clustering occurs in about 1-2% of cases, indicating a heritable component, while monozygotic twin concordance is low at around 4%, underscoring the role of non-genetic influences. Epigenetic modifications, such as and alterations, are also suspected to play a part in modulating relevant to disease onset, though their precise contributions require further elucidation. Environmental exposures represent key triggers, particularly occupational ones, with varying by and exposure type. Crystalline silica dust is a well-established , with ratios ranging from 3 to 5 in exposed individuals, often seen in or workers. Organic solvents (e.g., , ) and exposure have similarly been associated with elevated risk, with ratios of 2-3 reported in systematic reviews. Infections may also contribute, as evidenced by higher of DNA in SSc tissues, though no specific infectious agent has been confirmed as causative. Demographic factors further modulate risk, with females experiencing disease onset at a rate 4- to 10-fold higher than males, possibly linked to hormonal or X-chromosome influences. Incidence peaks between ages 30 and 50, though some cohorts report a slightly later onset around 45-64 years. Ethnic variations influence severity rather than incidence, with showing more diffuse cutaneous involvement and poorer prognosis compared to other groups.

Pathophysiological mechanisms

Systemic scleroderma, also known as systemic sclerosis (), is characterized by a complex interplay of vascular, immune, and fibrotic processes that drive disease progression. The canonical three-phase model describes the pathogenesis as an initial vascular phase involving endothelial damage, followed by an inflammatory phase with immune cell activation, and culminating in a fibroproliferative phase marked by excessive remodeling. This sequential progression underscores the interconnectedness of these mechanisms, where early vascular injury perpetuates immune dysregulation and across multiple organs. Recent studies as of 2025 have further elucidated subpopulations via single-cell analyses and potential contributions from gut alterations to immune-fibrotic crosstalk. The vascular phase initiates with endothelial cell (EC) injury, often triggered by or infectious agents, leading to EC and progressive microvascular loss. This is accompanied by intimal proliferation, where fibroproliferative changes thicken arterial walls, impairing and tone. Perivascular , involving infiltration of + and + T cells around apoptotic ECs, further exacerbates damage. Endothelin-1 (ET-1), a potent , is upregulated in this phase, promoting sustained and endothelial-to-mesenchymal transition (EndoMT), which contributes to vessel wall and ischemia. These vascular alterations form the foundation for tissue and set the stage for subsequent immune and fibrotic responses. Immune activation in the inflammatory phase features prominent T-cell infiltration into affected tissues, with increased + and + T cells producing pro-fibrotic cytokines such as IL-13 and IFN-γ. B cells contribute through dysregulated production and elevated IL-6 secretion, while reduced regulatory B cells fail to dampen inflammation. Key cytokines like transforming growth factor-β (TGF-β) and IL-6, derived from these immune cells, bridge inflammation to fibrosis by activating downstream signaling pathways such as SMAD and MAPK. This immune dysregulation sustains a Th2-skewed response, with IL-4 and IL-13 enhancing collagen synthesis and fibroblast recruitment. In the fibrotic phase, resident undergo activation and differentiation into , characterized by expression of alpha- actin (α-SMA) and excessive production of (ECM) components, particularly collagen types I and III. TGF-β is central here, inducing and inhibiting ECM degradation via matrix metalloproteinases. (PDGF) further amplifies this process by stimulating and cell , leading to persistent ECM accumulation. These mechanisms extend to multi-organ involvement; for instance, alveolar in (ILD) arises from similar activation and cytokine-driven ECM deposition in pulmonary tissues, while scleroderma renal crisis (SRC) stems from renal vasculopathy with intimal and ischemic .

Diagnosis

Clinical evaluation

Clinical evaluation of systemic scleroderma begins with a detailed and to identify characteristic features suggestive of the disease. Patients often report Raynaud's phenomenon as an initial symptom, which involves episodic color changes in the fingers or toes triggered by or stress, and it frequently precedes the formal by several years (median 2.8 years overall after Raynaud's onset, IQR 0.7–10.0 years; longer in limited cutaneous forms, e.g., median 4.6 years for women), with a median time of approximately 4.6 years from onset to overall, though it can extend to 8-10 years or more in limited cutaneous forms. Other key historical elements include the development of puffy fingers, often described as a non-pitting affecting the hands, followed by progressive skin tightening starting distally and potentially spreading proximally. Inquiry into family history is essential, as it represents the strongest known risk factor, with first-degree relatives having a 13- to 15-fold increased relative risk compared to the general population. Environmental exposures, such as silica dust or organic solvents, should also be explored, as occupational or environmental triggers are associated with disease onset in susceptible individuals. The physical examination focuses on assessing skin involvement and vascular changes. Skin thickness is evaluated using the modified Rodnan skin score (mRSS), a validated semiquantitative tool that grades firmness and tethering on a scale of 0 (normal) to 3 (sclerosed) across 17 body sites, yielding a total score from 0 to 51; higher scores correlate with more extensive fibrosis and poorer prognosis. Nailfold capillaroscopy, a non-invasive bedside technique, reveals microvascular abnormalities crucial for early suspicion of systemic scleroderma. Characteristic patterns include the "early" scleroderma pattern with giant capillaries greater than 50 micrometers, the "active" pattern featuring hemorrhages and capillary loss alongside giants, and the "late" pattern marked by severe avascularity and ramified/bushy capillaries, reflecting progressive vascular damage. Classification relies on the 2013 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) criteria, an additive scoring system where a total score of 9 or greater indicates definite systemic scleroderma; these criteria demonstrate 91% and 92% specificity. Skin thickening of the fingers extending proximal to the metacarpophalangeal joints alone suffices for (9 points). Absent this, points are assigned as follows:
ItemSubitemPoints
Skin thickeningPuffy fingers (scleroderma pattern)2
Sclerodactyly (distal to metacarpophalangeal joints but proximal to proximal interphalangeal joints)4
Fingertip lesions (digital tip ulcers or pitting scars)2
-2
Abnormal nailfold capillaries-2
Pulmonary arterial hypertension and/or Maximum extent on either2
Raynaud's phenomenon-3
No major updates to these criteria have occurred since for adult patients. Subtype assignment—limited cutaneous systemic scleroderma (lcSSc) versus diffuse cutaneous systemic scleroderma (dcSSc)—is determined primarily by the extent of involvement, with lcSSc limited to areas distal to the elbows and knees (plus face and neck) and dcSSc extending proximally to include the trunk; organ-specific features, such as rapid progression in dcSSc, further refine classification. includes localized forms like , which spares the fingers and lacks systemic features, and , characterized by deep tissue induration without Raynaud's or vascular changes. Early clinical is critical, as it enables timely and , thereby improving long-term outcomes and reducing irreversible organ damage.

Laboratory and imaging studies

Laboratory studies play a crucial role in the of systemic scleroderma (), particularly through testing. Antinuclear antibodies () are positive in up to 95% of patients, typically detected by with nucleolar or speckled patterns that support the when clinical features are present. Specific autoantibodies, such as anti-centromere, anti-topoisomerase I (Scl-70), and anti-RNA polymerase III, further classify disease subsets and are detailed in autoantibody-specific protocols. Inflammatory markers like (ESR) and (CRP) are often elevated during active disease phases, reflecting inflammation but lacking specificity for SSc. Currently, no single biomarker reliably quantifies , though emerging panels like the liver fibrosis (ELF) test show correlations with disease severity in research settings. Imaging modalities provide objective assessment of organ involvement in SSc. High-resolution computed tomography (HRCT) of the chest is essential for evaluating (ILD), commonly revealing ground-glass opacities, reticular patterns, and subpleural involvement, particularly in the lung bases. Transthoracic echocardiography with Doppler is recommended for screening pulmonary arterial (PAH), where a tricuspid regurgitant (TR) velocity exceeding 2.8 m/s prompts confirmatory right heart catheterization per established algorithms like DETECT. (MRI) aids in assessing skin thickening and muscle involvement, detecting , , and fascial changes with high sensitivity, often in patients with musculoskeletal symptoms. Functional tests complement laboratory and imaging findings to quantify . Pulmonary function tests (PFTs), including forced (FVC) and (DLCO), are routinely used to monitor lung involvement; reduced FVC indicates restrictive physiology in ILD, while isolated DLCO decline may signal early PAH or . The 6-minute walk test assesses exercise tolerance and functional capacity, correlating with cardiopulmonary status and prognosis in patients with pulmonary complications. Nailfold capillaroscopy evaluates microvascular changes, identifying patterns like giant capillaries and avascular areas that are highly specific for early vascular involvement. Esophageal manometry detects gastrointestinal dysmotility, often showing absent and low lower esophageal pressure in over 80% of cases. The European Scleroderma Trials and Research (EUSTAR) group, through its database of over 23,000 patients, endorses annual screening with these tools to detect subclinical organ involvement early. is rarely performed for diagnosis due to its invasiveness but, when indicated, reveals characteristic dermal fibrosis, collagen accumulation, and vascular alterations.

Management

Symptomatic and supportive care

Symptomatic and supportive care in systemic scleroderma focuses on alleviating symptoms, preventing complications, and enhancing through non-disease-modifying strategies. These interventions target common manifestations such as skin tightening, vascular issues, gastrointestinal dysfunction, and limitations, often involving a multidisciplinary including dermatologists, rheumatologists, and therapists. modifications, topical treatments, and pharmacological aids are employed to manage discomfort and maintain function, with evidence supporting their role in reducing symptom burden without addressing underlying . Skin care is essential to mitigate dryness, pruritus, and ulceration associated with . Daily application of fragrance-free, emollient moisturizers, such as petroleum-based ointments, helps maintain and reduce cracking, particularly after bathing. Topical corticosteroids or calcineurin inhibitors like may be used for localized or , applied sparingly to avoid . For digital ulcers, which affect up to 50% of patients and risk , local wound care involves gentle cleansing with saline, of necrotic if needed, and application of hydrocolloid or occlusive dressings to promote and protect against trauma. Compression or pneumatic gloves can reduce hand and improve circulation, aiding in ulcer prevention and flexibility. The endothelin has been shown to reduce new digital ulcer formation by approximately 48% in clinical trials, with observational data from the PROSIT study confirming lower ulcer incidence in treated Italian cohorts. Management of Raynaud's phenomenon, a hallmark vascular symptom, emphasizes both non-pharmacological and pharmacological approaches to minimize episodes. Lifestyle measures include avoiding cold exposure through layered clothing, hand warmers, and stress reduction techniques, which can decrease attack frequency by up to 50% in some patients. , particularly dihydropyridine agents like (starting at 10-30 mg daily), are first-line therapy, improving digital blood flow and reducing risk in systemic sclerosis. These agents work by relaxing vascular , with clinical trials demonstrating significant symptom relief in 60-70% of cases. Pain control and gastrointestinal symptom relief require cautious interventions due to scleroderma's multisystem effects. Nonsteroidal anti-inflammatory drugs (NSAIDs), such as ibuprofen or naproxen, may be used intermittently for or but should be administered judiciously to avoid exacerbating gastrointestinal issues, often with gastroprotective agents. For (), prevalent in over 90% of patients, inhibitors (PPIs) like omeprazole are standard, healing and reducing risk. Prokinetic agents, including (10-20 mg before meals), enhance esophageal and gastric motility, alleviating dyspepsia and regurgitation in up to 70% of responsive cases. Supportive measures further promote functional independence and nutritional health. Physical and occupational therapy programs, including stretching exercises and splinting, help prevent joint contractures and maintain hand mobility, with studies showing improved after 12 weeks of . Nutrition counseling addresses from intestinal dysmotility, recommending small, frequent meals, supplements, and high-calorie diets to prevent and deficiencies. Patients with face heightened aspiration pneumonia risk, warranting annual influenza and pneumococcal vaccinations per guidelines for immunocompromised individuals.

Immunomodulatory therapies

Immunomodulatory therapies target the dysregulated in systemic scleroderma (SSc), particularly in diffuse cutaneous SSc (dcSSc) where early aggressive intervention can halt fibrotic progression driven by B-cells, T-cells, and cytokines like IL-6. These agents, including immunosuppressants and biologics, are recommended for patients with progressive disease, especially (ILD) or skin involvement, with monitoring for infections and risks due to their immunosuppressive effects. The 2024 British Society for Rheumatology (BSR) guidelines prioritize mycophenolate mofetil (MMF) as first-line therapy for SSc-ILD over (CYC), with rituximab or as alternatives for refractory cases. The 2024 EULAR recommendations also endorse MMF as first-line for SSc-ILD, with rituximab and as options for refractory cases. Mycophenolate mofetil () is the preferred first-line immunomodulator for SSc-ILD, administered at 2-3 g/day orally, as it stabilizes forced vital capacity (FVC) and is better tolerated than alternatives. In the Scleroderma Lung Study II (SLS II), a double-blind randomized trial of 142 patients with SSc-ILD, 24 months of improved FVC by a mean of +2.19%, compared to +2.88% with , with no significant difference between the groups. The 2024 BSR guidelines endorse for early dcSSc with ILD, citing its long-term efficacy in slowing progression. is reserved for severe or rapidly progressive SSc-ILD unresponsive to , with pulses preferred over oral due to reduced toxicity. The Lung Study I (SLS I), a placebo-controlled in 158 patients, demonstrated modest short-term FVC improvement (+2.53% vs. -0.94% at 12 months) but no sustained benefit beyond 18 months, alongside risks of and infections. Per 2024 BSR recommendations, (0.5-1 g/m² IV monthly for 6-12 months) serves as a second-line option, with close monitoring for and hematologic adverse effects. Biologic agents like rituximab, a B-cell depleting , show promise in reducing skin fibrosis and stabilizing lung function in refractory . In the DESIRES trial, a phase 2/3 study of 76 patients, two courses of rituximab (1 g IV at weeks 0 and 4) improved mRSS by -6.30 points at 24 weeks versus -2.56 with , with sustained benefits up to 48 weeks and acceptable safety. The 2024 BSR guidelines suggest rituximab as rescue therapy added to for progressive skin or lung involvement (1B evidence). , an IL-6 receptor inhibitor, is effective for SSc-ILD by mitigating and , particularly in early dcSSc with elevated inflammatory markers. The faSScinate phase 2 trial in 87 patients found subcutaneous tocilizumab (162 mg weekly) reduced mRSS by -6.03 versus -4.12 with at 24 weeks (observed means), with benefits sustained at 48 weeks in the open-label extension and preserved FVC in ILD subsets, with a favorable safety profile. The 2024 BSR guidelines recommend tocilizumab (162 mg subcutaneously weekly) as first-line for ATA-positive early dcSSc or as add-on for ILD (1B ), noting risks. For SSc-associated arthritis, at 15-25 mg/week orally is commonly used to control and early skin involvement, with evidence from prospective studies showing mRSS reductions of 4-5 points at 6-12 months. A review of multiple trials confirms MTX's efficacy in improving joint symptoms and skin scores in early diffuse SSc, comparable to MMF for non-ILD manifestations. In severe, rapidly progressive dcSSc, autologous transplantation (ASCT) offers superior long-term outcomes over CYC by resetting the . The ASTIS trial, a randomized study of 156 patients, reported improved event-free survival (74% vs. 51% at 4 years) and overall survival (approximately 81% vs. 57% at median 5.8 years follow-up) with myeloablative ASCT (high-dose CYC and ATG conditioning) despite early treatment-related mortality (10%). The 2024 BSR guidelines support ASCT for selected young patients with life-threatening dcSSc (1A evidence), emphasizing multidisciplinary evaluation for risks like infections and relapse.

Renal

The primary treatment for scleroderma renal crisis (SRC) involves aggressive blood pressure control using (ACE) inhibitors, with preferred as the initial agent due to extensive clinical experience in this setting. Early initiation of ACE inhibitors can lead to blood pressure normalization in up to 90% of patients, significantly improving renal outcomes and one-year survival rates to approximately 76%. For patients progressing to end-stage renal disease, is the standard supportive measure, with potential for recovery and dialysis discontinuation in about 50% of cases when ACE inhibitors are continued. In high-risk patients, such as those positive for anti-RNA polymerase III antibodies, prophylactic low-dose ACE inhibitors may be considered to mitigate SRC risk, though evidence remains limited and controversial.

Pulmonary

For systemic sclerosis-associated interstitial lung disease (SSc-ILD), the antifibrotic agent nintedanib is approved to slow disease progression, as demonstrated in the SENSCIS trial where it reduced the annual rate of forced vital capacity (FVC) decline by 44% compared to placebo over 52 weeks. This treatment targets fibrotic processes in the lungs, helping to preserve lung function in patients with progressive ILD. For pulmonary arterial hypertension (PAH) classified as World Health Organization group 1, which frequently complicates SSc, phosphodiesterase-5 inhibitors like sildenafil improve exercise capacity and hemodynamics, while endothelin receptor antagonists (ERAs) such as bosentan, ambrisentan, and macitentan reduce pulmonary vascular resistance and are standard first-line therapies. Combination regimens of sildenafil and ERAs may further enhance outcomes in SSc-PAH by addressing multiple vascular pathways.

Gastrointestinal

Gastrointestinal involvement in systemic sclerosis often requires targeted interventions for motility disorders and complications like strictures. For esophageal strictures causing , (Botox) injections into the lower esophageal sphincter can provide symptomatic relief by relaxing hypertonic muscle, particularly in cases mimicking achalasia due to dysmotility. In pseudo-obstruction from small bowel hypomotility, enteral nutrition via tube feeding supports nutritional status and prevents , serving as a key adjunct when oral intake is inadequate. The somatostatin analog enhances intestinal motility by increasing the frequency and duration of migrating motor complexes, reducing bacterial overgrowth and diarrhea in affected patients, with short-term administration showing significant improvements in scleroderma-related cases.

Cardiac

Cardiac manifestations in systemic sclerosis, including arrhythmias and , are managed with standard cardiovascular therapies tailored to the specific complication. For conduction abnormalities and bradyarrhythmias leading to symptomatic issues like syncope, permanent implantation is indicated to restore rhythm stability, addressing fibrosis-related electrical disturbances common in SSc myocardium. In cases of systolic or diastolic , such as are used to alleviate fluid overload and , improving symptoms in advanced stages where myocardial involvement predominates. Lung transplantation represents a definitive option for end-stage SSc-ILD or PAH refractory to medical therapy, with five-year post-transplant survival rates approximately 50%, comparable to other indications despite higher perioperative risks.

Epidemiology

Incidence and prevalence

Systemic scleroderma, also known as systemic sclerosis (SSc), has a global incidence of approximately 1.4 cases per 100,000 person-years, with ranges typically reported from 1 to 5 per 100,000 annually across studies. Incidence rates are notably higher in women, estimated at 3 to 28 per 100,000 compared to 0.8 to 7 per 100,000 in men, reflecting a female-to-male ratio of about 5:1. Regional variations show higher incidence in (1.4–5.6 per 100,000) and (0.6–2.3 per 100,000) than in . The global prevalence of SSc is pooled at 17.6 per 100,000 individuals, generally ranging from 10 to 50 per 100,000 in most populations. Exceptionally high prevalence has been documented in specific groups, such as up to 469 per 100,000 among full-blooded in . Prevalence is elevated in (13.5–44.3 per 100,000) and (7.2–33.9 per 100,000) relative to , where rates are lower, for example, pooled estimates of about 3.8 per 100,000 in the region. Incidence and prevalence appear stable or increasing in regions like , , with recent data indicating a steady increase (average annual percent change of 3.94% for incidence), potentially due to enhanced diagnostic capabilities and aging populations. Global estimates from 2023 indicate approximately 1.47 million people affected worldwide, though underdiagnosis of mild cases likely underestimates the true burden.

Demographic characteristics

Systemic scleroderma exhibits a marked female predominance, with a female-to-male ratio ranging from 4:1 to 10:1 across various populations. This disparity is particularly pronounced during childbearing years, with peak onset between 30 and 50 years and a mean age at diagnosis of 45 to 47 years. Juvenile systemic scleroderma, defined as onset before age 16, is rare, accounting for 3% to 5% of all cases. Ethnic variations influence the typical patient profile, with higher prevalence observed among compared to other groups; affected individuals in this population more frequently present with the diffuse cutaneous subtype. The Choctaw Native American tribe demonstrates one of the highest reported risks for systemic scleroderma. In contrast, prevalence appears lower among Asian populations relative to those of African or descent. Socioeconomic factors contribute to disparities in the patient profile, including delayed among underserved groups due to barriers such as limited access to specialized and longer travel distances for low-income individuals. Occupational exposures, particularly in manual laborers involved in industries like or , are associated with increased risk, reflecting a demographic skew toward blue-collar workers. Pregnancy in women with systemic scleroderma is linked to a higher risk of disease flares, especially in those with active disease at conception or during the peripartum period.

Prognosis

Survival rates

Survival in systemic scleroderma (), also known as systemic sclerosis, has markedly improved over the decades, primarily due to advances in the of pulmonary arterial (PAH) and (ILD). Early studies from the reported 5-year survival rates of approximately 50-60%, with 10-year rates around 54%. More recent s indicate 5-year survival rates of 80-90% and 10-year rates of 60-70%, reflecting better screening and therapeutic interventions for cardiopulmonary complications. For instance, data from a large Canadian (1994-2004) showed overall 5-year survival of 90% and 10-year survival of 82%. As of 2024, multicenter studies report 5-year survival of 85.9% and 10-year survival of 71.7%. Survival varies significantly by SSc subtype. Patients with limited cutaneous SSc (lcSSc) exhibit higher long-term survival, with 10-year rates of 80-90%, compared to 50-70% for diffuse cutaneous SSc (dcSSc), where more extensive skin and organ involvement contributes to poorer outcomes. SSc sine scleroderma, lacking significant skin involvement, has survival rates similar to lcSSc, with up to 92% survival at 15 years in some analyses. In a multicenter study, 5-year survival was 95% for lcSSc versus 81% for dcSSc. The leading causes of death in are primarily SSc-related, accounting for 60-70% of fatalities. PAH contributes to approximately 26-30% of deaths, ILD to 25-35%, (SRC) to about 10%, and infections to 15%, often exacerbated by or gastrointestinal involvement. These cardiopulmonary and renal complications underscore the organ-specific burden, with pulmonary involvement now surpassing renal crises as the dominant mortality driver due to historical declines in SRC incidence following the introduction of ACE inhibitors. A 2014 meta-analysis of multiple studies reported cumulative survival from of 74.9% at 5 years and 62.5% at 10 years, with pulmonary causes predominant. Data from the Trials and (EUSTAR) database, encompassing over 11,000 patients, show 3-year survival of 89.3% with ongoing improvements from better screening. Recent advances in targeted therapies, such as those recommended in 2024 EULAR guidelines, continue to enhance by addressing fibrotic manifestations.

Prognostic factors

Prognostic factors in systemic scleroderma, also known as systemic sclerosis (), play a crucial role in risk stratification and predicting disease course and outcomes. These factors encompass clinical subtypes, profiles, involvement metrics, organ-specific complications, and demographic characteristics. Identification of these predictors allows for personalized monitoring and intervention strategies to mitigate adverse events. The diffuse cutaneous (dcSSc) subtype is associated with poorer compared to limited cutaneous (lcSSc), with a (HR) for mortality of 1.90 in meta-analyses of studies. Similarly, positivity for anti-topoisomerase I antibodies (ATA, also known as anti-Scl-70) confers increased risk, with an HR of 1.38 for . In contrast, (ACA) are linked to better outcomes, with an HR of 0.62. panels, including these specificities, enable personalization of by stratifying patients for organ involvement risks and disease progression patterns. Early rapid skin progression, defined by trajectories with a skin thickening progression rate exceeding approximately 30 units per year (corresponding to a ΔmRSS >20 in the first year), predicts higher morbidity and mortality, with adjusted HRs of 4.05 to 5.85 compared to stable trajectories. Elevated baseline modified Rodnan skin score (mRSS >20) further worsens prognosis, correlating with increased internal organ involvement and mortality risk, with each 5-point increase associated with an approximate HR of 1.2. Limited skin involvement, as seen in lcSSc, is conversely a favorable indicator, reflecting slower progression and improved long-term survival. Interstitial lung disease (ILD) with forced vital capacity (FVC) <70% predicted is a strong negative predictor, with an HR of 2.79 for mortality. Pulmonary arterial hypertension (PAH), particularly when confirmed by right heart catheterization, carries an even higher risk, with an HR of 5.27. Low baseline (DLCO <70%) also indicates poor , with an HR of 3.31. Demographic factors further influence outcomes: onset after age 60 years is associated with heightened mortality risk (HR 5.79 in cohorts), likely due to comorbidities and accelerated organ involvement. Male sex confers an HR of 1.87, and African American ethnicity is linked to worse , with an increased of mortality up to 1.8 compared to white patients, attributed to higher rates of diffuse disease and severe pulmonary complications.

History and society

Historical aspects

The condition now recognized as systemic scleroderma has roots in ancient descriptions of skin hardening. Around 400 BC, documented cases of "thickened skin" in his medical notes, marking one of the earliest references to cutaneous manifestations resembling the disease. In the , European physicians provided more detailed accounts that distinguished localized and systemic forms. Italian physician Giovambattista Fantonetti described localized scleroderma in 1836, introducing the term "scleroderma generale" to characterize the progressive skin induration in a young woman. British surgeon reported systemic cases in the 1890s, including acroscleroderma linked to Raynaud's phenomenon, emphasizing visceral involvement such as joint contractures and organ . Additional European reports from the 1860s, including Maurice Raynaud's 1862 thesis on episodic vascular spasms, highlighted the circulatory and multi-organ features of the condition. The brought greater recognition of systemic scleroderma as a distinct entity affecting multiple organs. In 1945, Robert H. Goetz coined the term "progressive systemic sclerosis" to underscore its widespread nature beyond the skin, based on pathological observations of visceral changes. The limited cutaneous variant, later termed (calcinosis, Raynaud's phenomenon, esophageal dysmotility, sclerodactyly, ), was initially described in 1910 by physicians Thibierge and Weissenbach, with the acronym formalized in 1964. Autoantibodies, including antinuclear antibodies associated with , were identified in the , paving the way for serological diagnostics. In the , scleroderma renal crisis emerged as a critical complication, previously often fatal but rendered more manageable through the introduction of inhibitors. Key diagnostic and therapeutic milestones further refined understanding and classification. The American College of Rheumatology (ACR) published preliminary criteria in 1980, requiring proximal skin thickening as a major criterion or two minor criteria such as digital pitting scars or bibasilar for classification. Early clinical trials of autologous transplantation in 2001 demonstrated efficacy in halting disease progression in severe diffuse cases, offering a novel immunomodulatory approach. The 2013 ACR/European League Against (EULAR) classification criteria enhanced sensitivity for early and limited disease by incorporating profiles and nailfold capillaroscopy into a weighted scoring system. These developments have transformed systemic scleroderma from a uniformly progressive and often lethal disorder to one with targeted interventions and improved survival.

Support and awareness

Patient support networks play a crucial role in addressing the emotional, educational, and practical challenges faced by individuals with systemic . In the United States, the National Scleroderma Foundation (NSF), established in 1998, offers a of local chapters, online forums, and in-person groups to foster community and provide peer-to-peer encouragement for and families. The NSF has also invested over $33 million in since its founding, enhancing resources through evidence-based programs. In the , Scleroderma and Raynaud's UK (SRUK) operates a dedicated , regional groups, and virtual meetings tailored to those affected by and related conditions like Raynaud's phenomenon. Internationally, the Scleroderma Trials and group (EUSTAR) incorporates a advisory arm to integrate lived experiences into and care initiatives across . Public awareness efforts aim to reduce diagnostic delays and stigma surrounding systemic scleroderma, a condition often misunderstood due to its rarity. June is designated as globally, with activities peaking on World Scleroderma Day, June 29, to educate the public on symptoms and the need for early intervention. Campaigns frequently emphasize Raynaud's phenomenon as a common entry point for diagnosis, encouraging individuals with cold-induced finger discoloration to seek medical evaluation for potential underlying scleroderma. Celebrity advocates, including comedian , who supported research in honor of his sister with the disease, and actress , whose mother was diagnosed in 2006, have amplified these messages through media appearances and fundraising events. Key resources focus on empowering patients with comprehensive and holistic care. Support organizations deliver materials on symptom recognition, progression, and self-management strategies, while addressing needs through counseling referrals and workshops on coping with chronic illness-related anxiety and isolation. Access to multidisciplinary clinics—integrating rheumatologists, pulmonologists, and psychologists—is promoted to ensure coordinated , particularly beneficial for the diverse demographic profile of patients, who are predominantly women. However, disparities persist, with robust networks and campaigns more prevalent in high-income countries, whereas low- and middle-income regions face limited access to specialized groups and educational tools due to resource constraints.

Research

Ongoing clinical trials

As of 2025, numerous clinical trials are investigating novel interventions for systemic scleroderma (also known as systemic sclerosis, ), with over 100 studies registered on focusing on various disease manifestations. These trials emphasize targeted therapies addressing key pathogenic pathways, such as immune dysregulation and , while leveraging patient registries for longitudinal data collection. Recent additions include the phase 1 trial of ADI-001, an allogeneic CD20-targeted CAR-T , which dosed its first patient in July 2025 to evaluate and in autoimmune diseases including . Additionally, the phase 2b CONQUEST platform trial (NCT06195072) is evaluating nerandomilast and amlitelimab for early active SSc-ILD. In the domain of (ILD), a prominent ongoing phase 3 trial is the DAISY study (NCT05925803), evaluating subcutaneous , a type I receptor inhibitor, in adults with active diffuse cutaneous . This multicenter, randomized, double-blind, placebo-controlled trial assesses efficacy in reducing fibrosis and lung function decline, with primary endpoints including changes in modified Rodnan score and forced ; results are anticipated in late 2025. The completed phase 2 faSScinate and focuSSced trials of , an inhibitor, demonstrated trends toward improved skin thickness, leading to its approval for SSc-ILD; long-term data from extensions (up to 2021) confirmed sustained stabilization. Vascular complications, including pulmonary arterial hypertension (PAH) and digital ulcers, are addressed in ongoing observational studies of , a selective IP receptor agonist, evaluating its role in reducing digital burden and improving PAH hemodynamics in SSc patients refractory to standard therapies. For digital ulcers specifically, the completed phase 2 pilot RISE trial (NCT02915835) of , a soluble stimulator, in combination with background vasodilators showed no significant reduction in ulcer burden at 16 weeks, though the main RISE-SSc long-term extension (from NCT02283762) indicated potential benefits in skin and lung outcomes up to 2023. Notable among innovative approaches is the RESET-SSc trial (NCT06328777), a phase 1/2 open-label study of rese-cel (CABA-201), a CD19-directed CAR-T , in moderate-to-severe . Early 2025 data from the first infused patients indicate significant reductions in modified Rodnan skin scores (up to 20-point improvements) and B-cell depletion, suggesting reset without ongoing . Complementing this, the SENSCIS-ON open-label extension (NCT03313180) continues , a , in SSc-ILD patients post-parent trial, demonstrating sustained forced stabilization over 148 weeks with manageable gastrointestinal adverse events. Patient registries play a crucial role in supporting these trials by providing real-world longitudinal data. The European Scleroderma Trials and Research (EUSTAR) group maintains a database of over 14,000 patients across more than 200 centers, facilitating cohort enrichment and outcome tracking for ILD and endpoints. Similarly, the Scleroderma Research Registry, involving 12 centers, collects biosamples and clinical metrics to inform trial design and prognostic modeling in diverse SSc populations.

Emerging therapies

Emerging therapies for systemic scleroderma target key pathogenic pathways such as immune dysregulation, complement activation, and , with over 20 agents in the pipeline as of 2025, emphasizing precision approaches guided by autoantibodies like anti-RNA polymerase III. These innovations aim to address unmet needs in , (ILD), and organ-specific complications, though challenges persist in standardizing trial endpoints such as modified Rodnan Skin Score (mRSS) and (FVC). CAR-T cell therapy, particularly CD19-targeted approaches that deplete B cells, has shown promise in resetting aberrant immune responses. In the phase 1/2 RESET-SSc trial, with rese-cel (rescabtagene autoleucel) led to an mRSS of approximately 8 points (from 42 to 34) in early diffuse cutaneous systemic sclerosis patients at 6 months, alongside improvements in nailfold capillaroscopy and potential for disease remission. Preliminary data suggest sustained skin softening and immune reconstitution, positioning CAR-T as a transformative option for severe cases. Complement inhibitors represent another frontier, particularly for scleroderma renal crisis () involving . Observational studies with , a , demonstrated reduced C5b-9 deposition and stabilization of renal function in refractory SRC cases, with early administration potentially mitigating life-threatening and kidney injury. , a C5a receptor , is under exploration for vasculitic overlaps in systemic sclerosis, showing efficacy in reducing inflammation in related autoimmune conditions. Anti-fibrotic agents directly counteract profibrotic signaling. The completed phase 2 trial of fresolimumab, a neutralizing against all three TGF-β isoforms, improved skin scores and in early diffuse systemic sclerosis, with decreased biomarkers of such as α-smooth muscle . Similarly, pamrevlumab (FG-3019), an anti-connective tissue growth factor () , attenuated skin in preclinical systemic sclerosis models and is being evaluated for ILD manifestations. Expansions in stem cell therapies include mesenchymal stem cells (MSCs) and autologous (HSCT). MSCs exhibit antifibrotic, immunosuppressive, and proangiogenic effects, with 2025 trials like UPSIDE assessing early HSCT to enhance long-term remission and reduce toxicity in severe systemic sclerosis. JAK inhibitors, such as , are in phase 2 development for ILD, demonstrating reduced fibrosis in preclinical models by inhibiting JAK1/2-mediated cytokine signaling.

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