Biomaterial Database

Multicenter Outcome of Hematopoietic Stem Cell Transplantation for Primary Immune Deficiency Disorders in India. 2020

Revathi Raj, and Fouzia N Aboobacker, and Satya Prakash Yadav, and Ramya Uppuluri, and Sunil Bhat, and Dharma Choudhry, and Vikas Dua, and Gaurav Kharya, and Neha Rastogi, and Mansi Sachdev, and Vipin Khandelwal, and Venkateswaran Swaminathan, and Atish Bakane, and Balasubramaniam Ramakrishnan, and Biju George

Department of Pediatric Hematology and Oncology, Apollo Cancer Institutes, Chennai, India.

Hematopoietic stem cell transplantation (HSCT) is the curative option for many primary immune deficiency disorders (PID). In the last 5 years, increased awareness, availability of diagnostics based on flow cytometry, genetic testing, improved supportive care, use of reduced toxicity conditioning, and success of haploidentical donor HSCT have improved access to HSCT for children with PID in India. We present results on children with PID who underwent HSCT across India and the factors that influenced outcome. We collected retrospective data on the outcome of HSCT for PID from seven centers. We analyzed the impact of the type of PID, conditioning regimen, time period of HSCT- before or after January 2016, graft versus host disease prophylaxis, cause of mortality and overall survival. A total of 228 children underwent HSCT for PID at a median age of 12 months (range, 1 to 220 months) with a median follow up of 14.4 months. Infants accounted for 51.3% of the cohort and the male female ratio was 3:1. SCID (25%) and HLH (25%) were the more frequent diagnoses. Matched family donor was available in 36.4% and 44.3% children had a haploidentical HSCT. Reduced and myeloablative conditioning regimens were used with 64% children receiving a treosulfan based conditioning regimen. Peripheral blood stem cells were the predominant graft source at 69.3%. The survival in infants (60.2%) was inferior to children aged over 1 year (75.7% p value = 0.01). Children with Wiskott Aldrich syndrome (74.3%) and chronic granulomatous disease (82.6%) had the best outcomes. The survival was superior in children receiving HSCT from a matched sibling (78%) versus an alternate donor HSCT (61% p value = 0.04). In the cohort transplanted after January 2016 survival improved from 26.8% to 77.5% (p value = 0.00). Infection remains the main cause of mortality at in over 50% children. The 5-year overall survival rate was 68%. Survival of children with PID undergoing HSCT in India has improved dramatically in last 5 years. Alternate donor HSCT is now feasible and has made a therapeutic option accessible to all children with PID.

UI	MeSH Term	Description	Entries
D007194	India	A country in southern Asia, bordering the Arabian Sea and the Bay of Bengal, between Burma and Pakistan. The capitol is New Delhi.	Republic of India
D007223	Infant	A child between 1 and 23 months of age.	Infants
D008297	Male		Males
D002648	Child	A person 6 to 12 years of age. An individual 2 to 5 years old is CHILD, PRESCHOOL.	Children
D002675	Child, Preschool	A child between the ages of 2 and 5.	Children, Preschool,Preschool Child,Preschool Children
D005260	Female		Females
D006086	Graft vs Host Disease	The clinical entity characterized by anorexia, diarrhea, loss of hair, leukopenia, thrombocytopenia, growth retardation, and eventual death brought about by the GRAFT VS HOST REACTION.	Graft-Versus-Host Disease,Homologous Wasting Disease,Runt Disease,Graft-vs-Host Disease,Disease, Graft-Versus-Host,Disease, Graft-vs-Host,Disease, Homologous Wasting,Disease, Runt,Diseases, Graft-Versus-Host,Diseases, Graft-vs-Host,Graft Versus Host Disease,Graft-Versus-Host Diseases,Graft-vs-Host Diseases
D006801	Humans	Members of the species Homo sapiens.	Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D000081207	Primary Immunodeficiency Diseases	Genetic immunologic deficiency diseases and syndromes due to mutations in genes involved in IMMUNITY generally characterized by an increased susceptibility to infectious diseases. They are often associated with AUTOIMMUNE DISEASE manifestations.	Congenital Immunodeficiency Disease,Congenital Immunodeficiency Diseases,Congenital Immunodeficiency Disorder,Congenital Immunodeficiency Disorders,Congenital Immunodeficiency Syndrome,Congenital Immunodeficiency Syndromes,Inherited Immunodeficiency Disease,Inherited Immunodeficiency Diseases,Inherited Immunodeficiency Disorder,Inherited Immunodeficiency Disorders,Inherited Immunodeficiency Syndrome,Inherited Immunodeficiency Syndromes,Primary Antibody Deficiencies,Primary Antibody Deficiency Disorder,Primary Antibody Deficiency Disorders,Primary Antibody Deficiency Syndrome,Primary Antibody Deficiency Syndromes,Primary Immune Deficiency,Primary Immune Deficiency Disease,Primary Immune Deficiency Diseases,Primary Immune Deficiency Disorder,Primary Immune Deficiency Disorders,Primary Immune Deficiency Syndrome,Primary Immune Deficiency Syndromes,Primary Immunodeficiency Disease,Primary Immunodeficiency Disorder,Primary Immunodeficiency Disorders,Primary Immunodeficiency Syndromes,Antibody Deficiency, Primary,Deficiency, Primary Immune,Immune Deficiency, Primary,Immunodeficiency Disease, Congenital,Immunodeficiency Disease, Inherited,Immunodeficiency Disease, Primary,Immunodeficiency Diseases, Congenital,Immunodeficiency Diseases, Inherited,Immunodeficiency Diseases, Primary,Immunodeficiency Disorder, Congenital,Immunodeficiency Disorder, Inherited,Immunodeficiency Disorder, Primary,Immunodeficiency Disorders, Congenital,Immunodeficiency Disorders, Inherited,Immunodeficiency Syndrome, Congenital,Immunodeficiency Syndrome, Inherited,Immunodeficiency Syndrome, Primary,Immunodeficiency Syndromes, Congenital,Immunodeficiency Syndromes, Inherited,Immunodeficiency Syndromes, Primary,Inherited Immunodeficiency Syndrome,Primary Antibody Deficiency,Primary Immune Deficiencies,Primary Immunodeficiency Syndrome
D000293	Adolescent	A person 13 to 18 years of age.	Adolescence,Youth,Adolescents,Adolescents, Female,Adolescents, Male,Teenagers,Teens,Adolescent, Female,Adolescent, Male,Female Adolescent,Female Adolescents,Male Adolescent,Male Adolescents,Teen,Teenager,Youths