Hematopoietic Stem Cell Transplantation for Primary Immunodeficiency Disorders: Experience from a Referral Center in India

ABSTRACT

Objective: To share experience of over 15 years in hematopoieticstem cell transplantation in children with primaryimmunodeficiency disorders.Design: Medical record review.Setting: A referral center for pediatric hemato-oncologicaldisorders.Participants: Children (<18 y) diagnosed to have primaryimmune deficiencies who underwent hematopoietic stem celltransplantation between 2002 and August 2017.Main outcome measures: Disease-free survival, morbidity andmortality.Results: 85 primary immunodeficiency disorder transplants wereperformed with engraftment noted in 80 (94%) transplants and anoverall survival of 67%. The conditioning regimen wasindividualized based on the underlying immune defect. Mixedchimerism was noted in 20% children with 56% (9/16) remainingdisease-free. Graft versus host disease was noted in 33 (39.2%)children with most seen in children with chronic granulomatousdisease. Severe combined immune deficiency transplants weremainly complicated by infections. Immune cytopeniascomplicated Wiskott Aldrich syndrome and Hemophagocyticlymphohistiocytosis transplants. 29.4% (25/85) childrenunderwent haploidentical transplant in our cohort with a survival of70% in this group. Infectious complications were the mostcommon cause of death.Conclusion: Primary immunodeficiency disorders are curable inIndia when transplanted in centers with experienced and trainedpediatric transplant physicians and intensivists