Haploidentical Hematopoietic Stem Cell Transplantation for Patients with Severe Aplastic Anemia—Single-Centre Experience

TL;DR

This study shares the experience of using haploidentical stem cell transplants for severe aplastic anemia when other treatments fail.

Contribution

The paper presents a novel clinical experience with haploidentical transplants for SAA in a limited patient cohort.

Findings

All four patients successfully engrafted with median neutrophil engraftment at 21 days.

Only one patient experienced grade 2 acute GVHD, and no chronic GVHD occurred.

All patients achieved complete donor chimerism by day 60 post-transplant.

Abstract

Severe aplastic anemia (SAA) is a life-threatening type of aplastic anemia for which allogeneic stem cell transplantation or immunosuppressive therapy are the principal treatment modalities. Only about 25–30% of patients have a matched sibling donor, and finding an unrelated donor in ethnic minorities is a challenge. The use of related haploidentical donor transplants in severe aplastic anemia is uncommon. We would like to report our experience with the first four patients who underwent haploidentical transplants for severe aplastic anemia. This is a retrospective study. We collected data from our transplant database of all haploidentical hematopoietic stem cell transplants for SAA from 1 January 2020 to 31 December 2021. The transplant protocol used was the Hopkins’ protocol. There were three patients who underwent haploidentical transplants as primary therapy for SAA. A fourth patient received a haploidentical transplant after immunosuppressive therapy failure. The median age of the patients at transplant was 24 y (range 20–29). All patients were engrafted. Neutrophil engraftment occurred at a median of 21 days (range 17–22). Any active infections resolved with the recovery of blood counts. The median hospitalization time was 27 days (range 22–41). Only one patient had grade 2 acute GVHD involving the skin. There was no chronic GVHD. All patients had complete lymphoid and myeloid donor chimerism on day 60. Based on our experience and the emerging literature, haplo-identical transplantation should be considered for select young patients with SAA who have low chances of responding to immunosuppressive therapy.