Outcomes of paroxysmal nocturnal hemoglobinuria in the pediatric age group in a resource‐constrained setting

• Published in: Pediatric blood & cancer Vol. 67; no. 4; pp. e27712 - n/a
• Main Authors: Halder, Rohan, Mishra, Priyanka, Aggarwal, Mukul, Mannivanan, Prabhu, Dhawan, Rishi, Seth, Tulika, Tyagi, Seema, Mahapatra, Manoranjan, Pati, Hara P., Saxena, Renu
• Format: Journal Article
• Language: English
• Published: United States Wiley Subscription Services, Inc 01-04-2020
• Subjects: androgen and steroids in PNH; Androgens; Aplastic anemia; Bone marrow transplantation; Cyclosporins; Globulins; Hematology; Oncology; Paroxysmal nocturnal hemoglobinuria; pediatric PNH; Pediatrics; PNH and thrombosis; Renal failure; renal failure in PNH; Stem cell transplantation; Stem cells; pediatric PNH; renal failure in PNH; androgen and steroids in PNH; PNH and thrombosis
• ISSN: 1545-5009; 1545-5017
• DOI: 10.1002/pbc.27712

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.