NOVEL-1st: an observational study to assess the safety and efficacy of nilotinib in newly diagnosed patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase in Taiwan.

Nilotinib has been approved for the treatment of Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). However, the real-world evidence of nilotinib in newly diagnosed untreated Ph+ CML-CP is limited in Taiwan. The NOVEL-1st study was a non-interventional, multi-center study collecting long-term safety and effectiveness data in patients with newly diagnosed and untreated Ph+ CML-CP receiving nilotinib. We enrolled 129 patients from 11 hospitals. Overall, 1,466 adverse events (AEs) were reported; among these, 151 were serious and 524 were nilotinib-related. Common hematological AEs were thrombocytopenia (31.0%), anemia (20.9%), and leukopenia (14.0%); common nilotinib-related AEs were thrombocytopenia (29.5%), anemia (14.7%), and leukopenia (12.4%). Early molecular response, defined as BCR-ABL ≤ 10% at Month 3, was seen in 87.6% of patients. By 36 months, the cumulative rates of complete hematologic response, complete cytogenetic response, major molecular response, molecular response 4.0-log reduction, and molecular response 4.5-log reduction were 98.5, 92.5, 85.8, 65.0, and 45.0%, respectively. Nilotinib is effective and well-tolerated in patients with newly diagnosed Ph+ CML-CP in the real-world setting. Long-term holistic care and a highly tolerable AE profile may contribute to good treatment outcomes in Ph+ CML-CP under first-line treatment with nilotinib.

Retinoic acid syndrome in patients following the treatment of acute promyelocytic leukemia with all-trans retinoic acid.

BACKGROUND: Retinoic acid syndrome (RAS) is a potentially lethal complication during all-trans retinoic acid (ATRA) treatment of acute promyelocytic leukemia (APL). The incidence and risk factors have been shown to vary in different series. In this study we want to establish the incidence of RAS in our hospital and try to elucidate factors that increase its risk. METHODS: We retrospectively analyzed 102 patients diagnosed with APL between August 1993 and December 2007 at Chang Gung Memorial Hospital, Taiwan. All patients received ATRA as an induction regimen with or without conventional chemotherapy. RESULTS: Eight of the 102 patients (7.8%) experienced RAS which developed after a median of 9 days (range: 2 to 23 days) of ATRA treatment. Respiratory distress and fever were the most common presentations, occurring in 7 of 8 patients (87.5%). Age, gender, morphological or molecular subtypes, an initial white blood cell (WBC) count of more than 10 x 10(9)/L and concurrent chemotherapy did not statistically attribute to the occurrence of RAS. One patient developed RAS manifesting with pulmonary hemorrhage but experienced a complete recovery after administration of high-dose dexamethasone. The RAS-related mortality was 12.5% (1 out of 8 patients). CONCLUSION: The incidence of RAS in this study was similar to those of other series with ATRA and concurrent chemotherapy. Age, gender, morphological or molecular subtypes, an initial leukocyte count of more than 10 x 10(9)/L or the presence of concurrent chemotherapy is not significantly associated with the occurrence of the RAS.