Abstract

BackgroundTo evaluate cyclosporine A (CSA)-related neurotoxicity after haploidentical hematopoietic stem cell transplantation (HID-HSCT) in children with hematopathy.MethodsThis retrospective case series study included children with hematopathy who underwent HID-HSCT at Fujian Medical University Union Hospital between February 2013 and January 2017.ResultsFifty-one children (39 males) were included in the study with a median age of 8 (range, 1.1–18) years. Seven patients (13.7%) developed CSA-related neurotoxicity after a median 38 (range, − 3 to 161) days from HID-HSCT. Hypertension (5/7, 71%) was the most common prodrome. Brain magnetic resonance imaging showed posterior reversible encephalopathy syndrome in six patients and atypical abnormalities in one patient. One patient died from grade IV graft-versus-host disease (GvHD) on day + 160, and six patients were alive at the last follow-up. Four patients (71.4%) achieved complete remission, while two patients developed secondary epilepsy and exhibited persistent MRI and electroencephalogram abnormalities at the 5-year follow-up. Hypertension after CSA was more common in patients with CSA-related neurotoxicity than in those without (71% vs. 11%, P = 0.002). Five-year overall survival did not differ significantly between patients with CSA-related neurotoxicity (85.7 ± 13.2%) and those without (65.8 ± 7.2%).ConclusionsThe incidence of CSA-related neurotoxicity in children with hematopathy undergoing HID-HSCT is relatively high.

Highlights

  • To evaluate cyclosporine A (CSA)-related neurotoxicity after haploidentical hematopoietic stem cell transplantation (HID-Hematopoietic stem cell transplantation (HSCT)) in children with hematopathy

  • We retrospectively examined the data of children who underwent HID-HSCT in order to describe the risk factors and long-term outcomes of CSA-related neurotoxicity

  • Clinical characteristics of the patients included in the analysis A total of 51 pediatric patients (39 males, 76.5%) with a median age of 8 years were included in this study

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Summary

Introduction

To evaluate cyclosporine A (CSA)-related neurotoxicity after haploidentical hematopoietic stem cell transplantation (HID-HSCT) in children with hematopathy. Wang et al Italian Journal of Pediatrics (2021) 47:83 such as cyclosporine A (CSA) or tacrolimus, together with an immunosuppressant, such as low-dose methotrexate or mycophenolate, alternative agents are available [6]. CSA is the cornerstone of GvHD prophylaxis. Neurotoxicity occurs in 5– 11% of patients receiving CSA as GvHD prophylaxis after HSCT [9,10,11,12,13]. CSA-related neurotoxicity usually resolves completely with dose reduction or drug withdrawal, but this can have major implications on clinical outcomes, in the face of ongoing GvHD. Little information is available regarding CSA-related neurotoxicity in children undergoing HID-HSCT

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