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

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.

journal_name

Pediatr Blood Cancer

journal_title

Pediatric blood & cancer

authors

Halder R,Mishra P,Aggarwal M,Mannivanan P,Dhawan R,Seth T,Tyagi S,Mahapatra M,Pati HP,Saxena R

doi

10.1002/pbc.27712

subject

Has Abstract

pub_date

2020-04-01 00:00:00

pages

e27712

issue

4

eissn

1545-5009

issn

1545-5017

journal_volume

67

pub_type

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