# Autologous stem cell transplant for severe, progressive juvenile systemic sclerosis **Authors:** Shaikha Alqahtani, Fabiana Cacace, Andrea Bauchat, Brittany Paige DePriest, Timothy Alan Driscoll, Carmem Bonfim, Joanne Kurtzberg, Nicole Larrier, Jeffery Dvergsten, Kris Michael Mahadeo PMC · DOI: 10.1093/stcltm/szag005 · Stem Cells Translational Medicine · 2026-03-23 ## TL;DR This paper reports that autologous stem cell transplant improved a teenager's severe, treatment-resistant juvenile systemic sclerosis. ## Contribution The study presents a case where stem cell transplantation led to clinical improvement in a refractory jSSc patient. ## Key findings - Autologous stem cell transplant was associated with clinical improvement in a 17-year-old with severe jSSc. - The patient showed positive outcomes despite having sub-optimal therapeutic options previously. - This case suggests stem cell transplantation may be a viable treatment for refractory juvenile systemic sclerosis. ## Abstract Juvenile systemic sclerosis (jSSc) is a rare, chronic, autoimmune disease in children/adolescents and is associated with significant morbidity, skin thickening/hardening (scleroderma), organ toxicity and sub-optimal therapeutic options. In this report, autologous stem cell transplantation is associated with clinical improvement in a 17-year-old with refractory jSSc. Graphical Abstract ## Linked entities - **Diseases:** scleroderma (MONDO:0005100) ## Full-text entities - **Genes:** RNPC3 (RNA binding region (RNP1, RRM) containing 3) [NCBI Gene 55599] {aka CPHD7, IGHD5, RBM40, RNP, SNRNP65}, CD34 [NCBI Gene 100051718], CMPK1 (cytidine/uridine monophosphate kinase 1) [NCBI Gene 51727] {aka CK, CMK, CMPK, UMK, UMP-CMPK, UMPK}, CRP (C-reactive protein) [NCBI Gene 1401] {aka PTX1} - **Diseases:** myopathy (MESH:D009135), organ dysfunction (MESH:D009102), autoimmune (MESH:D001327), knee pain (MESH:D046788), chronic (MESH:D002908), pulmonary arterial hypertension (MESH:D000081029), fibrosis (MESH:D005355), weight loss (MESH:D015431), fever (MESH:D005334), heart failure (MESH:D006333), hypertension (MESH:D006973), fatigue (MESH:D005221), skin thickening/hardening (MESH:D013585), inflammation (MESH:D007249), Apnea Hypopnea (MESH:D020181), dysphagia (MESH:D003680), Raynaud phenomenon (MESH:D011928), ILD (MESH:D017563), infection (MESH:D007239), cardiac involvement (MESH:D006331), renal crisis (MESH:D006030), malnutrition (MESH:D044342), toxicity (MESH:D064420), muscle weakness (MESH:D018908), OSA (MESH:C535586), involvement (MESH:C564676), rash (MESH:D005076), TBI (MESH:D012793), lung disease (MESH:D008171), hypopigmentation (MESH:D017496), contractures (MESH:D003286), Juvenile systemic sclerosis (MESH:D012595) - **Chemicals:** campath (MESH:D000074323), mycophenolate mofetil (MESH:D009173), methotrexate (MESH:D008727), Cyclophosphamide (MESH:D003520), steroid (MESH:D013256), carbon monoxide (MESH:D002248), prednisone (MESH:D011241), Tocilizumab (MESH:C502936), oxygen (MESH:D010100), plerixafor (MESH:C088327), fludarabine (MESH:C024352), amlodipine (MESH:D017311) - **Species:** Equus caballus (domestic horse, species) [taxon 9796], Homo sapiens (human, species) [taxon 9606] ## Full text _Full body text omitted from this summary view._ Fetch the complete paper as Markdown: https://tomesphere.com/paper/PMC13006201/full.md ## Figures 2 figures with captions in the complete paper: https://tomesphere.com/paper/PMC13006201/full.md ## References 21 references — full list in the complete paper: https://tomesphere.com/paper/PMC13006201/full.md --- Source: https://tomesphere.com/paper/PMC13006201