Practical guidelines for monitoring and management of coagulopathy following tisagenlecleucel CAR T-cell therapy.


Journal

Blood advances
ISSN: 2473-9537
Titre abrégé: Blood Adv
Pays: United States
ID NLM: 101698425

Informations de publication

Date de publication:
26 01 2021
Historique:
received: 22 06 2020
accepted: 06 12 2020
entrez: 26 1 2021
pubmed: 27 1 2021
medline: 15 5 2021
Statut: ppublish

Résumé

Cytokine release syndrome (CRS) is a systemic inflammatory response associated with chimeric antigen receptor T-cell (CAR-T) therapies. In severe cases, CRS can be associated with coagulopathy and hypofibrinogenemia. We present our global multicenter experience with CRS-associated coagulopathy after tisagenlecleucel therapy in 137 patients with relapsed or refractory B-cell acute lymphoblastic leukemia from the ELIANA and ENSIGN trials. These trials included clinical guidelines for fibrinogen replacement during CRS-associated coagulopathy. Hypofibrinogenemia requiring replacement was observed only in patients with severe CRS. A higher percentage of patients who required replacement were <10 years old, compared with those who did not require replacement. Twenty-three patients received replacement for hypofibrinogenemia (<1.5 g/L); 9 of them developed marked hypofibrinogenemia (<1 g/L). Very low fibrinogen levels (<1 g/L) were documented in patients before maximal CRS (n = 1), during maximal CRS (n = 7), and at CRS improvement (n = 1). Although hypofibrinogenemia was the most clinically significant coagulopathy, some patients also developed prolonged prothrombin time and activated partial thromboplastin time and increased international normalized ratio, further increasing the risk of bleeding. Hypofibrinogenemia was effectively managed using fibrinogen concentrate or cryoprecipitate replacement; severe (grade 4) bleeding events were rare (n = 2). CRS-associated coagulopathy with hypofibrinogenemia is manageable according to empiric guidelines of fibrinogen replacement for CAR-T trials. Fibrinogen concentrate should be used when cryoprecipitate is not reliably available. Monitoring fibrinogen levels in patients with moderate or severe CRS is essential for avoiding potentially fatal bleeding events. These trials were registered at www.clinicaltrials.gov as #NCT02435849 and #NCT02228096.

Identifiants

pubmed: 33496754
pii: S2473-9529(21)00076-8
doi: 10.1182/bloodadvances.2020002757
pmc: PMC7839371
doi:

Substances chimiques

Receptors, Antigen, T-Cell 0
Receptors, Chimeric Antigen 0
tisagenlecleucel Q6C9WHR03O

Banques de données

ClinicalTrials.gov
['NCT02435849', 'NCT02228096']

Types de publication

Journal Article Multicenter Study Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

593-601

Subventions

Organisme : NCI NIH HHS
ID : P01 CA214278
Pays : United States

Informations de copyright

© 2021 by The American Society of Hematology.

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Auteurs

Jochen Buechner (J)

Department of Pediatric Hematology and Oncology, Oslo University Hospital, Oslo, Norway.

Stephan A Grupp (SA)

Division of Oncology, Center for Childhood Cancer Research and Cancer Immunotherapy Program, Children's Hospital of Philadelphia, Philadelphia, PA.
Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA.

Hidefumi Hiramatsu (H)

Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan.

David T Teachey (DT)

Division of Oncology, Center for Childhood Cancer Research and Cancer Immunotherapy Program, Children's Hospital of Philadelphia, Philadelphia, PA.
Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA.

Susana Rives (S)

Department of Pediatric Hematology-Oncology and Institut de Recerca, Hospital Sant Joan de Déu Barcelona, Barcelona, Spain.

Theodore W Laetsch (TW)

Department of Pediatrics and Harold C. Simmons Comprehensive Cancer Center, University of Texas Southwestern Medical Center and Children's Health, Dallas, TX.

Gregory A Yanik (GA)

Department of Pediatrics, University of Michigan Medical Center, Ann Arbor, MI.

Patricia Wood (P)

Novartis Pharmaceuticals Corporation, East Hanover, NJ; and.

Rakesh Awasthi (R)

Novartis Pharmaceuticals Corporation, East Hanover, NJ; and.

Lan Yi (L)

Novartis Pharmaceuticals Corporation, East Hanover, NJ; and.

Andrea Chassot-Agostinho (A)

Novartis Pharmaceuticals Corporation, East Hanover, NJ; and.

Lamis K Eldjerou (LK)

Novartis Pharmaceuticals Corporation, East Hanover, NJ; and.

Barbara De Moerloose (B)

Department of Pediatric Hematology-Oncology and Stem Cell Transplantation, Ghent University Hospital, Ghent, Belgium.

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Classifications MeSH