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

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

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 3...

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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
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Summary: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.
Bibliography:Corrections added on 27 January 2020 after first online publication: Prabhu Manivannan, Seema Tyagi, and Hara P. Pati have been added as co‐authors and the order of authors has been updated.
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ISSN:1545-5009
1545-5017
DOI:10.1002/pbc.27712