1. Nilotinib first-line therapy in patients with Philadelphia chromosome-negative/BCR-ABL-positive chronic myeloid leukemia in chronic phase:ENEST1st sub-analysis
- Author
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Maria Liz Paciello Coronel, Beatrice Vincenzi, Ljubomir Petrov, Fausto Castagnetti, Delphine Rea, Andrzej Hellmann, Francis J. Giles, Andreas Hochhaus, Valentin Garcia-Gutierrez, Maria Asuncion Echeveste Gutierrez, Franҫois-Xavier Mahon, Luca Dezzani, Philipp le Coutre, Gianantonio Rosti, Norbert Gattermann, Nicholas C.P. Cross, Jeroen Janssen, CCA - Cancer Treatment and quality of life, Hematology, Hochhaus, Andrea, Mahon, Franois-Xavier, le Coutre, Philipp, Petrov, Ljubomir, Janssen, Jeroen J. W. M., Cross, Nicholas C. P., Rea, Delphine, Castagnetti, Fausto, Hellmann, Andrzej, Rosti, Gianantonio, Gattermann, Norbert, Coronel, Maria Liz Paciello, Gutierrez, Maria Asuncion Echeveste, Garcia-Gutierrez, Valentin, Vincenzi, Beatrice, Dezzani, Luca, and Giles, Francis J.
- Subjects
Male ,0301 basic medicine ,Cancer Research ,Myeloid ,Original Article – Clinical Oncology ,Fusion Proteins, bcr-abl ,Gastroenterology ,Antineoplastic Agent ,0302 clinical medicine ,hemic and lymphatic diseases ,Philadelphia Chromosome ,Hematology ,Chronic myeloid leukemia ,Myeloid leukemia ,General Medicine ,Middle Aged ,Leukemia ,medicine.anatomical_structure ,Oncology ,030220 oncology & carcinogenesis ,Female ,Human ,medicine.drug ,Adult ,medicine.medical_specialty ,Adolescent ,Anemia ,Philadelphia Chromosome Negative ,Protein Kinase Inhibitor ,Antineoplastic Agents ,Real-Time Polymerase Chain Reaction ,Philadelphia chromosome ,Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative ,Young Adult ,03 medical and health sciences ,Internal medicine ,medicine ,Humans ,Philadelphia chromosome negative/BCR-ABL positive ,Protein Kinase Inhibitors ,Aged ,ENEST1st ,business.industry ,Nilotinib ,medicine.disease ,Pyrimidines ,030104 developmental biology ,Pyrimidine ,business ,Multiplex Polymerase Chain Reaction - Abstract
PURPOSE: The ENEST1st sub-analysis presents data based on Philadelphia chromosome (Ph) status, i.e., Ph+ and Ph-/BCR-ABL1 + chronic myeloid leukemia.METHODS: Patients received nilotinib 300 mg twice daily, up to 24 months.RESULTS: At screening, 983 patients were identified as Ph+ and 30 patients as Ph-/BCR-ABL + based on cytogenetic and RT-PCR assessment; 76 patients had unknown karyotype (excluded from this sub-analysis). In the Ph-/BCR-ABL1 + subgroup, no additional chromosomal aberrations were reported. In the Ph+ subgroup, 952 patients had safety and molecular assessments. In the Ph-/BCR-ABL1 + subgroup, 30 patients had safety assessments and 28 were followed up for molecular assessments. At 18 months, the molecular response (MR) 4 rate [MR4; BCR-ABL1 ≤0.01% on International Scale (IS)] was similar in the Ph-/BCR-ABL1+ (39.3%) and Ph+ subgroups (38.1%). By 24 months, the cumulative rates of major molecular response (BCR-ABL1IS≤0.1%;), MR4, and MR4.5(BCR-ABL1IS≤0.0032%) were 85.7, 60.7, and 50.0%, respectively, in the Ph-/BCR-ABL1 + subgroup, and 80.3, 54.7, and 38.3%, respectively, in the Ph+ subgroup. In both Ph-/BCR-ABL1 + and Ph+ subgroups, rash (20 and 22%), pruritus (16.7 and 16.7%), nasopharyngitis (13.3 and 10.4%), fatigue (10 and 14.2%), headache (10 and 15.8%), and nausea (6.7 vs 11.4%) were frequent non-hematologic adverse events, whereas hypophosphatemia (23.3 and 6.8%), anemia (10 and 6.5%), and thrombocytopenia (3.3 and 10.2%) were the common hematologic/biochemical laboratory events.CONCLUSION: Based on similar molecular response and safety results in both subgroups, we conclude that Ph-/BCR-ABL1 + patients benefit from nilotinib in the same way as Ph+ patients.
- Published
- 2017