In persons with CIDP, auto-HSCT can be conducted fully on an outpatient basis and induces significant clinical responses: A prospective study in a single center

Abstract

BackgroundChronic inflammatory demyelinating polyneuropathy (CIDP) is one of the world's most common treatable neuropathy which usually responds to immunosuppressive treatment. Autologous hematopoietic stem cell transplantation (aHSCT) is an intense way of inducing immunosuppression. ObjectiveWe analyze the evolution of CIDP patients treated with aHSCT in our center. MethodsBetween 2018 and 2023, persons with CIDP were prospectively autografted employing the “Mexican method” to conduct grafts on an outpatient basis, employing cyclophosphamide 200 mg/Kg and rituximab 1000 mg. The protocol is registered in ClinicalTrials.gov identifier NCT02674217. ResultsIn our center 21 autologous transplant cases were completed in 2018–2023. Seven patients provided data to assess the efficacy of the procedure. Positive responses (stabilization and/or improvement) were observed in all seven patients: Five reported improvements in the Inflammatory Neuropathy Cause and Treatment (INCAT) score and one reported stabilization. In the Inflammatory Rasch-Built Overall Disability Scale (I-RODS) score. Median INCAT score was 5 (range 1–9), whereas median I-RODS score was 24 (range 11–29). Five patients (71%) reported improvement in the INCAT score, one reported stabilization and one informed worsening; concerning the I-RODS score 5 (71%) informed improvement, whereas two reported stabilization. ConclusionaHSCT conducted fully in an outpatient basis, employing the conditioning regimen of the “Mexican method” appears to be a feasible therapeutic option for persons with CIDP. Additional studies are needed to confirm these observations.