Haploidentical Stem Cell Transplantation in Children with Benign Disorders: Improved Survival and Cost-Effective Care Over 15 Years from a Single Center in India

Abstract

We present our experience in haploidentical stem cell transplantation (haplo SCT) in children with benign disorders. We performed a retrospective study where children aged up to 18 years diagnosed to have benign disorders and underwent haplo SCT from 2002 to September 2017 were included. Of the 54 children, the most common indications were Fanconi anaemia 12 (22%), severe aplastic anaemia 8 (14%) and primary immune deficiency disorders (PID) 25 (46%). Post-transplant cyclophosphamide (PTCy) was used in 41 (75.9%) and ex vivo T depletion in 13 (24.1%). Engraftment rates were 70% with acute graft versus host disease in 36% and cytomegalovirus reactivation in 55% children. There was a statistically significant difference found between survival with siblings as donors as compared to parents (p value 0.018). Overall survival was 60% which is the 1-year survival, with 68% survival among those with PIDs. Cytokine release syndrome was noted in 12/41 (29%) of children who received T replete graft and PTCy. In children over 6 months of age, PTCy at a cost of INR 1200 provides cost effective T cell depletion comparable with TCR a/b depletion priced at INR 1200,000. Haplo SCT is feasible option for cure in children with benign disorder.

Haploidentical transplantation in pediatric non‐malignant diseases: A retrospective analysis on behalf of the Spanish Group for Hematopoietic Transplantation (GETH)

“…Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) is a feasible cure option for children with malignant and nonmalignant diseases (NMDs) such as bone marrow failure syndromes, inborn errors of metabolism, and hemoglobinopathies [1][2][3][4] where no matched related or unrelated donor is available. Haplo-HSCT has achieved an engraftment rate of 70%, a durable graft rate of 67%, and an overall survival (OS) rate of 60%.…”

“…Haplo-HSCT has achieved an engraftment rate of 70%, a durable graft rate of 67%, and an overall survival (OS) rate of 60%. 4 The advantages of haploidentical donors include immediate and almost universal availability, avoidance of unrelated donor search costs, and availability for potential post-transplantation cellular therapy. 5,6 Recent data from the European Society for Blood and Marrow Transplantation indicated a 22% and 24.5% increase in allogeneic HSCT for primary immunodeficiency disorders (PIDs) and inborn errors of metabolism and stabilization for thalassemia.…”

“…15,20 Compared with matched sibling donors, haplo-HSCT in NMDs has a reduced OS. 21,22 Various studies have reported a 1-or 2-year OS rate of 60-80% in an NMD group using TCD or PT-Cy with low rates of GvHD and improved immune reconstitution using an ex vivo αβ + TCD platform and B-cell depletion, 4,23 and haplo-HSCT PT-Cy. [24][25][26][27][28][29] However, data on NMDs remain scarce and limited to disease groups and procedures, thereby necessitating a collaborative effort to understand the outcome for this patient group.…”

Haploidentical transplantation in pediatric non-malignant diseases: A retrospective analysis on behalf of the Spanish Group for Hematopoietic Transplantation (GETH)

“…Haploidentical stem cell transplantation (haplo-HSCT) is a feasible option for a cure in children with both malignant and non-malignant diseases (NMDs) such as bone marrow failure syndromes, inborn errors of metabolism (IEM) and hemoglobinopathies [1][2][3][4] where no matched related or unrelated donor is available. This procedure has achieved engraftment rates of 70%, durable graft of 67% and overall survival (OS) of 60% 4 .…”