Outcomes after intensive chemotherapy for secondary and myeloid-related changes acute myeloid leukemia patients aged 60 to 75 years old: a retrospective analysis from the PETHEMA registry.

Treatment options for patients with secondary acute myeloid leukemia (sAML) and AML with myeloid-related changes (AMLMRC) aged 60 to 75 years are scarce and unsuitable. A pivotal trial showed that CPX-351 improved complete remission with/without incomplete recovery (CR/CRi) and overall survival (OS) as compared with standard "3+7" regimens. We retrospectively analyze outcomes of 765 patients with sAML and AML-MRC aged 60 to 75 years treated with intensive chemotherapy, reported to the PETHEMA registry before CPX-351 became available. The CR/CRi rate was 48%, median OS was 7.6 months (95% confidence interval [CI]: 6.7-8.5) and event-free survival (EFS) 2.7 months (95% CI: 2-3.3), without differences between intensive chemotherapy regimens and AML type. Multivariate analyses identified age ≥70 years, Eastern Cooperative Oncology Group performance status ≥1 as independent adverse prognostic factors for CR/CRi and OS, while favorable/intermediate cytogenetic risk and NPM1 were favorable prognostic factors. Patients receiving allogeneic stem cell transplant (HSCT), autologous HSCT, and those who completed more consolidation cycles showed improved OS. This large study suggests that classical intensive chemotherapy could lead to similar CR/CRi rates with slightly shorter median OS than CPX-351.

Beclometasone dipropionate: a topically active corticosteroid for the treatment of gastrointestinal graft-versus-host disease.

BACKGROUND: Acute graft-versus-host disease (GVHD) is one of the most severe complications following allogeneic transplantation and the involvement of the gut has been associated with increased mortality and a poorer response to transplant. The use of systemic corticosteroids remains the standard first-line treatment, despite their severe secondary effects. OBJECTIVE: Beclometasone dipropionate (BDP) is a topically active corticosteroid with low absorption into the systemic circulation, which minimises many of the deleterious side effects associated with systemic corticosteroids. METHODS/RESULTS: Phase II and III trials evaluating the efficacy of BDP were reviewed. In the Phase II trials, 77% of patients with gastrointestinal GVHD who received BDP as a single agent responded and 50% did not require systemic corticosteroids, thus avoiding prolonged exposure to prednisone. Randomised trials demonstrated that BDP is safe and effective in treating acute gastrointestinal GVHD when used with a short induction course of prednisone, reducing the risk of GVHD treatment failure by > 60% and reducing mortality one year after randomisation by 45%. CONCLUSION: These results provide a particularly strong rationale for the incorporation of steroid-sparing regimens such as oral BDP in acute GVHD treatment.