Ramya Uppuluri1, Dhaarani Jayaraman2, Meena Sivasankaran2, Shivani Patel2, Venkateswaran Vellaichamy Swaminathan2, Lakshman Vaidhyanathan3, Sathishkumar Kandath4, Revathi Raj2. 1. Department of Pediatric Haematology, Oncology, Blood and Marrow Transplantation, Apollo Cancer Institutes, Chennai, India. Correspondence to: Dr Ramya Uppuluri, Department of Pediatric Hematology, Oncology, Blood and Marrow Transplantation, Apollo Cancer Institutes, 320, Padma Complex, Anna Salai, Teynampet, Chennai 600 035, India. ramya.december@gmail.com. 2. Department of Pediatric Haematology, Oncology, Blood and Marrow Transplantation, Apollo Cancer Institutes, Chennai, India. 3. Department of Haematology and Stem Cell Pheresis, Apollo Cancer Institutes, Chennai, India. 4. Department of Pediatric Critical Care, Apollo Cancer Institutes, Chennai, India.
Abstract
OBJECTIVE: To share experience of over 15 years in hematopoietic stem cell transplantation in children with primary immunodeficiency disorders. DESIGN: Medical record review. SETTING: A referral center for pediatric hemato-oncological disorders. PARTICIPANTS: Children (<18 y) diagnosed to have primary immune deficiencies who underwent hematopoietic stem cell transplantation between 2002 and August 2017. MAIN OUTCOME MEASURES: Disease-free survival, morbidity and mortality. RESULTS: 85 primary immunodeficiency disorder transplants were performed with engraftment noted in 80 (94%) transplants and an overall survival of 67%. The conditioning regimen was individualized based on the underlying immune defect. Mixed chimerism was noted in 20% children with 56% (9/16) remaining disease-free. Graft versus host disease was noted in 33 (39.2%) children with most seen in children with chronic granulomatous disease. Severe combined immune deficiency transplants were mainly complicated by infections. Immune cytopenias complicated Wiskott Aldrich syndrome and Hemophagocytic lymphohistiocytosis transplants. 29.4% (25/85) children underwent haploidentical transplant in our cohort with a survival of 70% in this group. Infectious complications were the most common cause of death. CONCLUSIONS: Primary immunodeficiency disorders are curable in India when transplanted in centers with experienced and trained pediatric transplant physicians and intensivists.
OBJECTIVE: To share experience of over 15 years in hematopoietic stem cell transplantation in children with primary immunodeficiency disorders. DESIGN: Medical record review. SETTING: A referral center for pediatric hemato-oncological disorders. PARTICIPANTS: Children (<18 y) diagnosed to have primary immune deficiencies who underwent hematopoietic stem cell transplantation between 2002 and August 2017. MAIN OUTCOME MEASURES: Disease-free survival, morbidity and mortality. RESULTS: 85 primary immunodeficiency disorder transplants were performed with engraftment noted in 80 (94%) transplants and an overall survival of 67%. The conditioning regimen was individualized based on the underlying immune defect. Mixed chimerism was noted in 20% children with 56% (9/16) remaining disease-free. Graft versus host disease was noted in 33 (39.2%) children with most seen in children with chronic granulomatous disease. Severe combined immune deficiency transplants were mainly complicated by infections. Immune cytopenias complicated Wiskott Aldrich syndrome and Hemophagocytic lymphohistiocytosis transplants. 29.4% (25/85) children underwent haploidentical transplant in our cohort with a survival of 70% in this group. Infectious complications were the most common cause of death. CONCLUSIONS:Primary immunodeficiency disorders are curable in India when transplanted in centers with experienced and trained pediatric transplant physicians and intensivists.
Authors: Daniel Leung; Gilbert T Chua; Alric V Mondragon; Youjia Zhong; Le Nguyen-Ngoc-Quynh; Kohsuke Imai; Pandiarajan Vignesh; Narissara Suratannon; Huawei Mao; Wen-I Lee; Yae-Jean Kim; Godfrey C F Chan; Woei Kang Liew; Le Thi Minh Huong; Hirokazu Kanegane; Dina Muktiarti; Xiaodong Zhao; Fatima Johanna Santos-Ocampo; Amir Hamzah Abdul Latiff; Reinhard Seger; Hans D Ochs; Surjit Singh; Pamela P Lee; Yu Lung Lau Journal: Front Immunol Date: 2020-08-13 Impact factor: 7.561