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Reduced‐toxicity alternate‐donor stem cell transplantation with posttransplant cyclophosphamide for primary immunodeficiency disorders
Author(s) -
Rastogi Neha,
Katewa Satyendra,
Thakkar Dhwanee,
Kohli Shruti,
Nivargi Sagar,
Yadav Satya Prakash
Publication year - 2018
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.26783
Subject(s) - medicine , treosulfan , thiotepa , thymoglobulin , fludarabine , alemtuzumab , busulfan , cyclophosphamide , transplantation , hematopoietic stem cell transplantation , primary immunodeficiency , total body irradiation , graft versus host disease , gastroenterology , surgery , tacrolimus , chemotherapy , disease
We describe here the outcomes of reduced‐toxicity alternate‐donor stem cell transplant (SCT) with posttransplant cyclophosphamide (PTCy) for primary immunodeficiency disorders (PIDs) in eight children (haploidentical—seven and matched unrelated donor—one). The conditioning was with serotherapy (alemtuzumab‐3/rabbit‐anti‐thymoglobulin‐5); fludarabine, cyclophosphamide, and total body irradiation‐5 (additional thiotepa‐3); fludarabine and treosulfan‐2; and fludarabine and busulfan‐1. All received PTCy 50 mg/kg on days 3 and 4 as graft versus host disease prophylaxis along with tacrolimus and mycophenolate. Mean CD34 dose was 13.8 × 10 6 /kg. Two children died because of PIDs. Acute graft versus host disease up to grades I and II was seen in three children. All six survivors are fully donor and disease free at median follow‐up of 753 days. Alternate donor SCT with PTCy is feasible in PID and has good outcomes.