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Outcomes of paroxysmal nocturnal hemoglobinuria in the pediatric age group in a resource‐constrained setting
Author(s) -
Halder Rohan,
Mishra Priyanka,
Aggarwal Mukul,
Mannivanan Prabhu,
Dhawan Rishi,
Seth Tulika,
Tyagi Seema,
Mahapatra Manoranjan,
Pati Hara P.,
Saxena Renu
Publication year - 2020
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.27712
Subject(s) - paroxysmal nocturnal hemoglobinuria , medicine , eculizumab , bone marrow failure , aplastic anemia , hemoglobinuria , hematopoietic stem cell transplantation , stem cell , anemia , gastroenterology , pediatrics , transplantation , bone marrow , immunology , haematopoiesis , antibody , complement system , biology , genetics
Abstract Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal stem cell disorder. Eculizumab and bone marrow transplantation are disease‐modifying treatments for PNH but may not be readily available in resource‐constrained settings. Of 52 pediatric patients with PNH, 20 had classical PNH and 32 had PNH/aplastic anemia (PNH/AA). Median time to diagnosis was 30 months in classical PNH patients. Renal failure was present in four patients (20%). Six (30%) achieved complete response, 10 (50%) achieved partial response with androgens in classical PNH. Two underwent allogenic stem cell transplantation. In the PNH/AA group, 16 (50%) were in CR and seven (21%) were in PR with anti‐thymocyte globulin ± cyclosporine.