Systemic sclerosis & Raynaud's
Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation for Diffuse Cutaneous Systemic Sclerosis: Identifying Disease Risk Factors for Toxicity and Long‐Term Outcomes in a Prospective, Single‐Arm Trial
Authors' conclusion
We observed favorable OS and EFS after AHSCT for patients with SSc, using CY200/ATG, unmanipulated PBSCs, and MMF posttransplant maintenance, which was comparable to trials with CD34+ graft selection.
Study context
- Population
- Patients with high-risk diffuse systemic sclerosis (SSc)
- Participants
- 20 patients
- Outcome
- OS and EFS after AHSCT
- Follow-up
- 5 years
Main finding
As reported in the abstract
Overall survival (OS) point estimate
85%
95% CI 60.4%–94.9%
Event-free survival (EFS) point estimate
75%
95% CI 50%–88.7%
Observational data: associations, not effects.
Further findings
Median follow-up for living patients
- Median follow-up after transplant for living patients (range 5.6-11.6)
- 7.5 years
Relapse/progression of SSc after AHSCT
- Patients developing relapse/progression of SSc after AHSCT
- 5
Anti-RNA polymerase III antibody–positive patients with prior scleroderma renal crisis and lowest-quartile estimated glomerular filtration rate (eGFR) at entry · early after transplant
- Patients among nine anti-RNA polymerase III–positive with prior scleroderma renal crisis and lowest-quartile eGFR
- 4
- Of these four, all developed prolonged organ failure/death early after transplant
- 4
Early transplant-related mortality
- Patients with early transplant-related mortality
- 2 (10%)
Safety
ICU treatment required early after transplant
- Patients requiring ICU treatment
- 8 (40%)
Original publication
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