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.

Sorafenib for hepatocellular carcinoma patients beyond Milan criteria after orthotopic liver transplantation: a case control study.

BACKGROUND: Orthotopic liver transplantation (OLT) is one of the most effective treatments for patients with hepatocellular carcinoma (HCC) within the Milan criteria. However, for patients beyond these criteria, the recurrence rate is higher and the prognosis is worse. Sorafenib is the only drug showing survival benefits in advanced HCC patients; however, its role in patients beyond the Milan criteria after OLT remains unclear and requires further investigation. METHODS: As a case-control study, we retrospectively analyzed 17 Chinese patients beyond Milan criteria undergoing OLT for HCC. These patients were stratified into adjuvant (n = 5), palliative (n = 6), and control groups (n = 6). RESULTS: Nine of 11 patients who received sorafenib after OLT needed dose reduction due to more than grade 2 side effects. The disease-free survival rates for patients with or without adjuvant sorafenib were 100% versus 37.5% (p = 0.034) at 6 months, 66.7% versus 9.4% (p = 0.026) at 12 months, and 66.7% versus 0.0% (p = 0.011) at 18 months, respectively. The overall survival rates for patients in palliative and control groups were 66.7% versus 40.0% (p = 0.248) at 6 months, 66.7% versus 40.0% (p = 0.248) at 12 months, and 50.0% versus 20.0% (p = 0.17) at 18 months, respectively. Patients in the adjuvant group had better overall survival rates than those in the palliative and control groups (p = 0.031) at 24-month follow-up. CONCLUSIONS: Adjuvant sorafenib could possibly extend both disease-free and overall survival for HCC patients beyond Milan criteria after OLT.