Long-Term Follow-Up in Patients With Chronic Myeloid Leukemia Treated With Ponatinib in a Real-World Cohort: Safety and Efficacy Analysis.
Clin Lymphoma Myeloma Leuk
; 24(3): 158-164, 2024 03.
Article
em En
| MEDLINE
| ID: mdl-37973457
ABSTRACT
BACKGROUND:
Ponatinib is a third-generation tyrosine-kinase inhibitor (TKI), indicated in patients with chronic phase (CP), accelerated phase (AP), or blast phase (BP) chronic myeloid leukemia (CML), who are resistant or intolerant to ≥2 prior TKIs, patients for whom subsequent treatment with imatinib is not appropriate, and patients who have a T315I mutation. PATIENTS ANDMETHODS:
We aimed to evaluate outcomes of ponatinib treatment, including safety, with focus on cardiovascular toxicity, in real-world patients from Argentina. Data from patients with CP CML treated with ponatinib was retrospectively retrieved from 2013 to 2023 in 7 centers.RESULTS:
Seventy-two patients were included (median age 44 years; male 55.5%; T315I mutation 32% median treatment duration 36 months. At baseline, 57 patients (79%) had a breakpoint cluster region-Abelson (BCRABL1) transcript level >10% on the international reporting scale (BCRABL1 IS). A molecular response (MR, BCRABL1 (IS) <1%) was achieved at 12 months in 51.6% of evaluable patients; 57% maintained MR at last follow-up. Overall, 43% and 25% maintained major MR (MMR) or deep MR (DMR) (MR4.0-MR5.0), respectively at last follow-up. Twelve (16.6%) ponatinib-resistant patients were rescued with allogeneic hematopoietic stem cell transplantation. The estimated 2-year progression-free survival (PFS) was 84%. Ponatinib dose was reduced during treatment in 22 patients; nevertheless, MMR was maintained in 50% of these patients. Severe arterial occlusive events (AOE) were reported in 10.9% of patients after a median treatment of 5 months.CONCLUSION:
CV toxicity was consistent with clinical trials and other real-world registries. Older age, hypercholesterolemia and a SCORE risk >2% were significantly associated with higher risk of AOEs. Controlling CV risk factors and reducing doses at optimal time points may help to optimize ponatinib use in daily practice.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Piridazinas
/
Leucemia Mielogênica Crônica BCR-ABL Positiva
/
Imidazóis
/
Antineoplásicos
Limite:
Adult
/
Humans
/
Male
Idioma:
En
Ano de publicação:
2024
Tipo de documento:
Article