1. Autologous hematopoietic stem cell transplantation improves long-term survival-data from a national registry
- Author
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Norbert, Blank, Marc, Schmalzing, Pia, Moinzadeh, Max, Oberste, Elise, Siegert, Ulf, Müller-Ladner, Gabriela, Riemekasten, Claudia, Günther, Ina, Kötter, Gabriele, Zeidler, Christiane, Pfeiffer, Aaron, Juche, Ilona, Jandova, Jan, Ehrchen, Laura, Susok, Tim, Schmeiser, Cord, Sunderkötter, Jörg H W, Distler, Margitta, Worm, Alexander, Kreuter, Gernot, Keyßer, Hanns-Martin, Lorenz, Thomas, Krieg, Nicolas, Hunzelmann, and Jörg, Henes
- Subjects
Male ,Scleroderma, Systemic ,Scleroderma, Diffuse ,Hematopoietic Stem Cell Transplantation ,Humans ,Female ,Registries ,Transplantation, Autologous ,Retrospective Studies - Abstract
Background Current recommendations on the management of systemic sclerosis (SSc) suggest that autologous hematopoietic stem cell therapy (HSCT) can be a rescue therapy for patients with rapidly progressive SSc. Objectives To assess the safety and efficacy of HSCT for patients with SSc and to compare these with non-HSCT patients in a control cohort with adjusted risk factors. Methods A retrospective analysis of data from the multicentric German network for systemic scleroderma (DNSS) with 5000 patients with SSc. Control groups consisted of all patients with diffuse cutaneous (dc)-SSc (group A) and an adjusted high-risk cohort of male patients with Scl70-positive dc-SSc (group B). Results Eighty SSc patients received an HSCT 4.1 ± 4.8 years after SSc diagnosis. Among them, 86.3% had dc-SSc, 43.5% were males, and 71.3% were positive for Scl70 antibodies. The control group A (n=1513) showed a significant underrepresentation of these risk factors for mortality. When the survival of the control group B (n=240) was compared with the HSCT group, a lower mortality of the latter was observed instead. Within 5 years after HSCT, we observed an improvement of the mRSS from 17.6 ± 11.5 to 11.0 ± 8.5 (p=0.001) and a stabilization of the DLCO. We did not see differences in transplant-related mortality between patients who received HSCT within 3 years after SSc diagnosis or later. Conclusion Our analysis of real-life data show that the distribution of risk factors for mortality is critical when HSCT cohorts are compared with non-HSCT control groups.
- Published
- 2022