Real-World Evidence of Crizanlizumab Showing Reductions in Vaso-Occlusive Crises and Opioid Usage in Sickle Cell Disease.

Autor: DeBonnett L; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA., Joshi V; Novartis Healthcare Pvt. Ltd., Hyderabad, India., Silva-Pinto AC; Department of Medical Imaging, Hematology and Oncology, Ribeirão Preto School of Medicine, University of São Paulo, Sao Paulo, Brazil., Colombatti R; Azienda Ospedale-Università di Padova, Padova, Italy., Pasanisi A; Centro Microcitemia-UOC, Ematologia Con Trapianto, Brindisi, Italy., Arcioni F; SC Pediatric Onco-Hematology With Bone Marrow Transplantation, Azienda Ospedaliera di Perugia, Perugia, Italy., Cançado RD; Department of Hematology/Oncology, Hospital Samaritano, Sao Paulo, Brazil., Sarp S; Novartis Pharma AG, Basel, Switzerland., Sarkar R; Novartis Healthcare Pvt. Ltd., Hyderabad, India., Soliman W; Novartis Saudi Ltd., Riyadh, Saudi Arabia.
Jazyk: angličtina
Zdroj: European journal of haematology [Eur J Haematol] 2024 Oct 29. Date of Electronic Publication: 2024 Oct 29.
DOI: 10.1111/ejh.14323
Abstrakt: Objective: Access to crizanlizumab, a disease-modifying therapy for sickle cell disease (SCD), was provided through a managed access program (MAP, NCT03720626). The present analysis evaluated the impact of 12 months of crizanlizumab treatment on vaso-occlusive crises (VOCs), and on the use of opioids for VOC-related pain relief, in patients with SCD from the MAP.
Methods: From June 2018 to January 2023, 112 patients with a history of recurrent VOCs completed 12 months of crizanlizumab (5 mg/kg) treatment and were monitored for adverse events (AEs).
Results: Crizanlizumab led to reductions of 18.0% and 36.2% in the proportions of patients having ≥ 1 home- and ≥ 1 healthcare-managed VOCs. Median absolute changes (interquartile range) from baseline in the rates of home- and healthcare-managed VOCs were -3.0 (-6.0, -1.0) and -2.0 (-4.0, 0), respectively. Data stratified by genotype and prior hydroxyurea use showed similar reductions in VOC rates. A 35.5% reduction in the proportion of patients requiring opioids was noted. AEs were consistent with earlier reports, and no new safety concerns were identified.
Conclusions: Crizanlizumab demonstrated potential benefits in attenuating VOC episodes, irrespective of SCD genotype and prior hydroxyurea use, and in lowering opioid usage. The safety of crizanlizumab was consistent with earlier reports.
Trial Registration: The MAP has been registered at ClinicalTrials.gov with the ID, NCT03720626.
(© 2024 Novartis and The Author(s). European Journal of Haematology published by John Wiley & Sons Ltd.)
Databáze: MEDLINE
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