Diagnosis dan Tatalaksana Paroxysmal Nocturnal Hemoglobinuria
DOI:
https://doi.org/10.55175/cdk.v48i7.93Keywords:
ravulizumab, paroxysmal nocturnal hemoglobinuria (PNH), hemolisis intravaskular, EculizumabAbstract
Paroxysmal nocturnal hemoglobinuria (PNH) merupakan kelainan sel hematopoesis didapat yang menyebabkan hemolisis intravaskular dan berisiko tinggi trombosis. PNH terdiri dari PNH klasik, PNH terkait kegagalan sumsum tulang, dan PNH subklinis. Diagnosis PNH dengan flow cytometry (FCM). Terapi PNH saat ini menggunakan eculizumab, ravulizumab, dan transplantasi sel punca alogenik.
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired disorder of hematopoietic stem cell causing intravascular hemolysis and high risk of thrombosis. It can be differentiated into: classical PNH, PNH associated with bone marrow failure, and subclinical PNH. Diagnosis is made by flow cytometry. PNH is treated with eculizumab, ravulizumab, and allogenic stem cell transplantation.
Downloads
References
Père NM, Lenaerts T, Pacheco JM, Dingli D. Evolutionary dynamics of paroxysmal nocturnal hemoglobinuria. PLOS Computational Biology 2018;14(6):e1006133. https://doi.org/10.1371/journal.pcbi.1006133
Devalet B, Mullier F, Chatelain B, Dogne JM, Chatelain C. Pathophysiology, diagnosis, and treatment of paroxysmal nocturnal hemoglobinuria: A review. Eur J Haematol 2015;95(3):190-8.
Parker CJ. Management of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitory therapy. Hematol Am Soc Hematol Educ Program 2011;2011(1):21– 9.
Borowitz MJ, Craig FE, DiGiuseppe JA, Illingworth AJ, Rosse W. Sutherland DR, et al. Guidelines for the diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria and related disorders by flow cytometry. Clin Cytometry 2010;78B(4).
Weitz IC. Thrombosis in patients with paroxysmal nocturnal hemoglobinuria. Semin Thromb Hemost. 2011;37(3):315-21.
de Latour RP, Mary JY, Salanoubat C, Terriou L, Etienne G, Mohty M, et al. Paroxysmal nocturnal hemoglobinuria: Natural history of disease subcategories. Blood 2008;112(8):3099–106.
Parker CJ. Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria. Hematology Am Soc Hematol Educ Program 2016;2016(1):208-16.
Lima M, Laboratory studies for paroxysmal nocturnal hemoglobinuria, with emphasis on flow cytometry. Pract Lab Med. 2020;20:e00158.
Hill A, Kelly RJ, Hillmen P. Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood 2013;121(25):4985– 96.
Richards SJ, Whitby L, Cullen MJ, Dickinson AJ, Granger V, Reilly JT, et al. Development and evaluation of a stabilized whole-blood preparation as a process control material for screening of paroxysmal nocturnal hemoglobinuria by flow cytometry. Cytometry B Clin Cytom. 2009;76B(1):47–55.
Sutherland DR, Keeney M, Illingworth A. Practical guidelines for the high-sensitivity detection and monitoring of paroxysmal nocturnal hemoglobinuria clones by flow cytometry. Cytometry B Clin Cytom. 2012;82B(4):195–208.
Pu JJ, Brodsky RA. Paroxysmal nocturnal hemoglobinuria from bench to bedside. Clin Transl Sci. 2011;4(3):219–24.
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(12):3897–902.
Sugimori C, Chuhjo T, Feng X, Yamazaki H, Takami A, Teramura M, et al. Minor population of CD55-CD59- blood cells predicts response to immunosuppressive therapy and prognosis in patients with aplastic anemia. Blood 2006;107(4):1308–14.
Malato A, Saccullo G, Coco LL, Mancuso S, Santoro M, Martino S, et al. Thrombotic complications in paroxysmal nocturnal haemoglobinuria: A literature review. Blood Transfus. 2012;10(4):428–35.
Ziakas PD, Poulou LS, Rokas GI, Bartzoudis D, Voulgarelis M. Thrombosis in paroxysmal nocturnal hemoglobinuria: Sites, risks, outcome. J Thromb Haemost. 2007;5(3):642-5.
Araten DJ, Thaler HT, Luzzatto L. High incidence of thrombosis in African-American and Latin-American patients with Paroxysmal Nocturnal Haemoglobinuria. J Thromb Haemost. 2005;93(1):88–91.
Raghupathy R, Derman O. Response of paroxysmal nocturnal hemoglobinuria clone with aplastic anemia to rituximab. Case Rep Hematol. 2012;2012:106182.
Stern RM, Connell NT. Ravulizumab: A novel C5 inhibitor for the treatment of paroxysmal nocturnal hemoglobinuria. Ther Adv Hematol. 2019;10:1-11.
Loschi M, Porcher R, Barraco F, Terriou L, Mohty M, Guibert S, et al. Impact of eculizumab treatment on paroxysmal nocturnal hemoglobinuria: A treatment versus no-treatment study. Am J Hematol. 2015;91(4):366-70.
Schubert J, Hillmen P, Roth A, Young NS, Elebute MO, Szer J, et al. Eculizumab, a terminal complement inhibitor, improves anaemia in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2008;142(2):263–72.
Brodsky RA, Young NS, Antonioli E, Risitano AM, Schrezenmeier H, Schubert J, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood 2008;111(4):1840–7.
Roth A, Hock C, Konik A, Christoph S, Duhrsen U. Chronic treatment of paroxysmal nocturnal hemoglobinuria patients with eculizumab: Safety, efficacy, and unexpected laboratory phenomena. Int J Hematol. 2011;93:704–14.
Kelly RJ, Hill A, Arnold LM, Brooksbank GL, Richards SJ, Cullen M, et al. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: Sustained efficacy and improved survival. Blood 2011;117(25):6786–92.
de Latour RP, Schrezenmeier H, Bacigalupo A, Blaise D, de Souza CA, Vigouroux S, et al. Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria. Haematologica 2012;97(11):1666–73.
Füreder W, Sperr WR, Heibl S, Zebisch A, Pfeilstocker M, Stefanzl G, et al. Prognostic factors and follow-up parameters in patients with paroxysmal nocturnal hemoglobinuria (PNH): Experience of the Austrian PNH network. Ann Hematol 2020;99: 2303-13.
Downloads
Published
How to Cite
Issue
Section
License
Copyright (c) 2021 Cermin Dunia Kedokteran
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
