Outcomes of paroxysmal nocturnal hemoglobinuria in the pediatric age group in a resource-constrained setting.
PNH and thrombosis
androgen and steroids in PNH
pediatric PNH
renal failure in PNH
Journal
Pediatric blood & cancer
ISSN: 1545-5017
Titre abrégé: Pediatr Blood Cancer
Pays: United States
ID NLM: 101186624
Informations de publication
Date de publication:
04 2020
04 2020
Historique:
received:
31
10
2018
revised:
09
02
2019
accepted:
15
02
2019
pubmed:
27
3
2019
medline:
27
6
2020
entrez:
27
3
2019
Statut:
ppublish
Résumé
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.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e27712Informations de copyright
© 2019 Wiley Periodicals, Inc.
Références
Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995;333:1253-1258.
Socié G, Mary JY, de Gramont A, et al. Paroxysmal nocturnal haemoglobinuria: long-term follow- up and prognostic factors-French Society of Haematology. Lancet. 1996;348:573-577.
Ware RE, Hall SE, Rosse WF. Paroxysmal nocturnal hemoglobinuria with onset in childhood and adolescence. N Engl J Med. 1991;325:991-996.
Miyata T, Yamada N, Iida Y, et al. Abnormalities of PIG-A transcripts in granulocytes from patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1994;330:249-255.
Takeda J, Miyata T, Kawagoe K, et al. Deficiency of the GPI anchor caused by a somatic mutation of the PIG-A gene in paroxysmal nocturnal hemoglobinuria. Cell. 1993;73:703-711.
Hall C, Richards S, Hillmen P. Primary prophylaxis with warfarin prevents thrombosis in paroxysmal nocturnal hemoglobinuria (PNH). Blood. 2003;102:3587-3591.
de Latour RP, Mary JY, Salanoubat C, et al. Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories. Blood. 2008;112(8):3099-3106.
Hill A, DeZern AE, Kinoshita T, et al. Paroxysmal nocturnal hemoglobinuria. Nat Rev Dis Primers. 2017;3:17028.
Borowit MJ, Crag FE, Digiuseppe JA, et al. Guidelines for the diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria and related disorders by flow cytometry. Cytometry B Clin Cytom. 2010;78(4):211-230.
Endo M, Beatty PG, Vreeke TM, Wittwer CT, Singh SP, Parker CJ. Syngeneic bone marrow transplantation without conditioning in a patient with paroxysmal nocturnal hemoglobinuria: in vivo evidence that the mutant stem cells have a survival advantage. Blood. 1996;88:742-750.
Graham ML, Rosse WF, Halperin EC, Miller CR, Ware RE. Resolution of Budd-Chiari syndrome following bone marrow transplantation for paroxysmal nocturnal haemoglobinuria. Br J Haematol. 1996;92:707-710.
Flotho C, Strahm B, Kontny U, et al. Stem cell transplantation for paroxysmal nocturnal haemoglobinuria in childhood. Br J Haematol. 2002;118:124-127.
Miller DR, Baehner RL, Diamond LK. Paroxysmal nocturnal hemoglobinuria in childhood and adolescence: clinical and erythrocyte metabolic studies in two cases. Pediatrics. 1967;39:675-688.
Kletzel M, Arnold WC, Berry DH. Paroxysmal nocturnal hemoglobinuria presenting as recurrent hemolytic uremic syndrome. Clin Pediatr. 1987;26:319-320.
Lin HC, Chen RL, Wang PJ. Paroxysmal nocturnal hemoglobinuria presenting as moyamoya syndrome. Brain Dev. 1996;18:157-159.
Rizk S, Ibrahim IY, Mansour IM, Kandil D. Screening for paroxysmal nocturnal hemoglobinuria (PNH) clone in Egyptian children with aplastic anemia. J Trop Pediatr. 2002;48:132-137.
Wyatt HA, Mowat AP, Layton M. Paroxysmal nocturnal haemoglobinuria and Budd-Chiari syndrome. Arch Dis Child. 1995;72:241-242.
Wainwright L, Brodsky RA, Erasmus LK, Poyiadjis S, Naidu G, MacKinnon D. Paroxysmal nocturnal hemoglobinuria arising from Fanconi anemia. J Pediatr Hematol Oncol. 2003;25:167-168.
van den Heuvel-Eibrink MM, Bredius RG, te Winkel ML, et al. Childhood paroxysmal nocturnal haemoglobinuria (PNH), a report of 11 cases in the Netherlands. Br J Haematol. 2005;128:571-577.
Naithani R, Mahapatra M, Dutta P, Kumar R, Pati HP, Choudhry VP. Paroxysmal nocturnal hemoglobinuria in childhood and adolescence-a retrospective analysis of 18 cases. Indian J Pediatr. 2008;75:575-578.
Curran KJ, Kernan NA, Prockop SE, et al. Paroxysmal nocturnal hemoglobinuria in pediatric patients. Pediatr Blood Cancer. 2012;59:525-529.
Young NS, Maciejewski JP, Sloand E, et al. The relationship of aplastic anemia and PNH. Int J Hematol. 2002;76(Suppl. 2):168-172.
Wang H, Chuhjo T, Yasue S, Omine M, Nakao S. Clinical significance of a minor population of paroxysmal nocturnal hemoglobinuria-type cells in bone marrow failure syndrome. Blood. 2002;100:3897-3902.
Narita A, Muramatsu H, Okuno Y, Sekiya Y. Development of clinical paroxysmal nocturnal hemoglobinuria in children with aplastic anemia. Br J Haematol. 2017;178:954-958.
Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124:2804-2811.
Bourantas K. High-dose recombinant human erythropoietin and low dose corticosteroids for treatment of anemia in paroxysmal nocturnal hemoglobinuria. Acta Haematol. 1994;91:62-65.
Marwaha RK, Bansal D, Trehan A, Varma N. Androgens in childhood acquired aplastic anemia in Chandigarh, India. Tropical Doctor. 2004;34:149-152.
Andolin JR, Reinis AL, Akhta R, et al. Successful reduced-intensity conditioning hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria with aplastic anemia in two children. Pediatr Blood Cancer. 2018;65(8):e27218.