Expert consensus on the management of pharmacodynamic breakthrough-hemolysis in treated paroxysmal nocturnal hemoglobinuria.

Autor: Dingli D; Mayo Clinic, Rochester, MN, USA., De Castro Iii C; Duke University Medical Center, Duke Cancer Institute, Durham, NC, USA., Koprivnikar J; Hackensack University Medical Center, Hackensack, NJ, USA., Kulasekararaj A; King's College Hospital and King's College London, London, England., Maciejewski J; Cleveland Clinic, Cleveland, OH, USA., Mulherin B; Hematology Oncology of Indiana, PC, American Oncology Network, Indianapolis, IN, USA., Panse J; RWTH Aachen University Hospital, Aachen, Germany + Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf (CIO ABCD), Aachen, Germany., Pullarkat V; City of Hope, Duarte, CA, USA., Röth A; West German Cancer Center, University Hospital Essen, University of Duisburg-Essen, Essen, Germany., Shammo J; Northwestern University Feinberg School of Medicine, Chicago, IL, USA., Terriou L; University Hospital of Lille, Lille, France., Weitz I; Keck School of Medicine at the University of Southern California, Los Angeles, CA, USA., Yermilov I; PHAR, Beverly Hills, CA, USA., Gibbs S; PHAR, Beverly Hills, CA, USA., Broder M; PHAR, Beverly Hills, CA, USA., Beenhouwer D; PHAR, Beverly Hills, CA, USA., Kuter D; Massachusetts General Hospital, Boston, MA, USA.
Jazyk: angličtina
Zdroj: Hematology (Amsterdam, Netherlands) [Hematology] 2024 Dec; Vol. 29 (1), pp. 2329030. Date of Electronic Publication: 2024 Mar 21.
DOI: 10.1080/16078454.2024.2329030
Abstrakt: Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, non-malignant hematologic disease characterized by complement-mediated hemolysis (with or without hemoglobinuria), fatigue, increased susceptibility to thrombosis, and bone marrow dysfunction. The development of complement inhibitors has transformed outcomes for patients with PNH, but patients may still experience pharmacodynamic breakthrough hemolysis (BTH), which can be caused by exposure to a complement amplifying condition (CAC), such as vaccination, infection, or surgery.
Materials and Methods: A 13-member expert panel used a validated methodology (a RAND/UCLA modified Delphi panel) to develop consensus on how to classify pharmacodynamic BTH in patients with complement-inhibitor treated PNH. Physicians reviewed literature, rated the appropriateness of over 400 scenarios, and discussed the ratings at an in-person meeting.
Results: After the meeting, the panel agreed on 77% of scenarios. Here, we present the group's agreed-upon recommendations on how to manage BTH caused by a CAC, as well as provide a severity classification system for BTH and strategies to mitigate risk of BTH in special circumstances (e.g. vaccination, planned or unplanned surgery, and pregnancy).
Discussion: In general, as severity of BTH increased, experts agreed more interventions to manage the BTH were appropriate. These recommendations are based on clinical experience and opinion. Without clear data from randomized trials to guide the management of BTH, expert opinion can be useful to support patient care.
Databáze: MEDLINE
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