Nilotinib is an orally administered BCR-ABL tyrosine kinase inhibitor that has shown good clinical efficacy in imatinib-resistant or -intolerant, Philadelphia chromosome-positive, chronic myeloid leukaemia (CML) in a phase I/II trial. The phase I component of the trial established the dosage regimen used in the phase II part of the trial, which included several arms. Three of these arms, or phase II trials, evaluated nilotinib in each of the three phases of CML (chronic, accelerated or blast crisis).I n the phase II trial in patients with chronic-phase CML, major cytogenetic response (primary endpoint) was achieved in 48% of the 280 patients who received nilotinib and had a follow-up period of > or = 6 months. Major cytogenetic response rates did not differ between imatinib-resistant and -intolerant patients, and nilotinib was effective in patients with BCR-ABL mutations (except T315I). Haematologic response rate (primary endpoint) was 47% in the phase II trial with nilotinib in patients with accelerated-phase CML (n = 119). Complete haematologic response was achieved in 26% of patients and 21% had no evidence of leukaemia or returned to chronic-phase CML. Major cytogenetic response, an important secondary endpoint in the trial, occurred in 29% of patients. Data from the phase II trial in patients with CML in blast crisis (n = 135) also showed promising results, with 39% of patients achieving haematologic response with nilotinib. Adverse events reported with nilotinib have generally been of mild to moderate severity. Grade 3 or 4 neutropenia and thrombocytopenia were reported in 29% of patients each.
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http://dx.doi.org/10.2165/00003495-200868040-00005 | DOI Listing |
Breast
March 2025
Department of Oncology, Sahlgrenska University Hospital, Sweden; Institute of Clinical Sciences, Sahlgrenska Academy, University of Gothenburg, Sweden.
Clin Ther
March 2025
Fondazione IRCCS Istituto Neurologico Carlo Besta, Medical Genetics and Neurogenetics Unit, Milan, Italy. Electronic address:
Purpose: The 6 months pilot, single arm, phase I/II, open-label clinical trial PHEMI investigated the safety and efficacy of daily administration of phenylbutyrate in reducing lactic acidosis by at least 20% in 3 children (ages 7-10 yrs) with pyruvate dehydrogenase deficiency and 6 adults with mitochondrial myopathy encephalopathy lactic acidosis and stroke-like episodes. As a side study, we investigated the response to phenylbutyrate treatment in skin fibroblasts and cybrids derived from PHEMI patients with the aim of unraveling a possible in vivo-in vitro correlation.
Methods: Safety was assessed through the collection of vital signs, clinical evaluations, blood samples, and reported adverse events.
Clin Lymphoma Myeloma Leuk
February 2025
Memorial Sloan Kettering Cancer Center, New York, NY. Electronic address:
Background: In the phase 3 CANDOR trial (NCT03158688), daratumumab added to carfilzomib and dexamethasone (KdD) significantly prolonged progression-free survival relative to carfilzomib and dexamethasone (Kd) alone in previously treated patients with relapsed/refractory multiple myeloma (RRMM).
Materials And Methods: We present a post hoc analysis of the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30-item module (EORTC QLQ-C30) and EORTC QLQ Myeloma 20-item module (EORTC QLQ-MY20) patient-reported outcome (PRO) measures from the CANDOR trial.
Results: Median (range) duration of observation for PROs was 18.
Am J Kidney Dis
March 2025
Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam, Netherlands. Electronic address:
Photodiagnosis Photodyn Ther
March 2025
Department of Urology, Keio University Hospital, Tokyo, Japan.
Background: This analysis utilized a subject-based analysis of data from the prospective single-arm phase III trial (jRCT2061210055) to evaluate the efficacy of photodynamic diagnosis (PDD) when 5-aminolevulinic acid hydrochloride (5-ALA) was administered 4-8 h before transurethral resection of bladder tumors (TURBT).
Methods: This study evaluated 144 patients suspected of having non-muscle invasive bladder cancer (NMIBC), including 127 with NMIBC (38 patients had normal-to-flat-appearing cancer) and 17 without cancer. The diagnostic accuracy and clinical utility of blue light (BL) during TURBT were assessed for each patient and compared with those of white light (WL).
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