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Ibrutinib is a small molecule inhibitor of Bruton's tyrosine kinase indicated for the treatment of relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), and mantle cell lymphoma (MCL). The Named Patient Program in Australia and New Zealand (ANZ NPP) provided access to ibrutinib treatment to 1126 R/R CLL/SLL and 330 R/R MCL patients, prior to Pharmaceutical Benefits Scheme listing. This study aimed to assess the duration of treatment for the ANZ NPP patients, as an indicator of efficacy and tolerability of ibrutinib in the real world. Based on the NPP data, ibrutinib provided a median of 47 months clinical benefit for participants with CLL/SLL and 14 months clinical benefit for those with MCL; outcomes that are consistent with the clinical trial results and further support the well-established efficacy and safety profile of ibrutinib in the real world.

Myelodysplastic syndromes (MDS) have a major impact on quality of life (QoL). We performed a post hoc analysis of two multicenter trials of azacitidine-based disease-modifying therapy for patients with MDS and low blast count acute myeloid leukemia (AML), to identify factors associated with QoL. 231 patients were included (median age 70 years). At baseline, higher initial hemoglobin, but not neutrophil or platelet count, was associated with better global QoL and physical function ( p  < 0.001 and p  = 0.001, respectively). During therapy, increase in hemoglobin was associated with improvement in QoL and physical function ( p  = 0.005 and p  < 0.001, respectively). Lower initial hemoglobin was associated with higher dyspnea and fatigue scores ( p  < 0.001 and p  = 0.001, respectively), and hemoglobin response was associated with improvement in dyspnea and fatigue ( p  < 0.001 for each). In patients with MDS and low blast count AML, hemoglobin level was strongly correlated with global QoL, physical functioning, dyspnea and fatigue, both before and during azacitidine-based therapy.

Venetoclax, a potent B-cell lymphoma-2 (BCL-2) inhibitor, has demonstrated clinical efficacy in chronic lymphocytic leukemia (CLL). VENICE II is an open-label, single-arm, phase 3b study (NCT02980731) evaluating the impact of venetoclax monotherapy (400 mg once daily) for ≤2 years on health-related quality of life (HRQoL) of patients with relapsed/refractory CLL. The primary endpoint was mean change in the global health status (GHS)/quality of life (QoL) subscale of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) from baseline to Week 48. Overall, 210 patients received ≥1 dose of venetoclax; median treatment duration was 67.4 weeks. The primary endpoint was met with mean improvement of +9.3 points ( n  = 156, 95% confidence interval 6.1-12.5; p =.004) in GHS/QoL. At Week 48, clinically meaningful improvements were observed for role functioning, fatigue, and insomnia domains of EORTC QLQ-C30, suggesting venetoclax monotherapy has a positive impact on HRQoL. No new safety signals were reported.

The efficacy of paracetamol (acetaminophen) as an antipyretic during febrile neutropenia (FN) has not previously been established. We conducted a randomized double-blind placebo-controlled feasibility trial: hemato-oncology patients at high FN risk were randomly assigned to six hourly oral paracetamol (1 g) or placebo during the first 42 hours of FN. Fifty-three participants were screened, thirty-seven enrolled; 22 developed FN and commenced treatment (13 paracetamol; 9 placebo); recruitment rates were below, and retention rates met, pre-defined feasibility criteria. During the first 24 hours of FN, paracetamol recipients had significantly lower peak temperature than placebo: mean 38.2 (standard deviation 0.8) °C versus 38.9 (0.4) °C; difference -0.78 °C (95% CI -1.38 to -0.18); p  = .013. Bacterial load measurement was not informative. Paracetamol lowers body temperature during FN, and definitive trials to determine its impact on FN outcomes are needed. Australian New Zealand Clinical Trials Registry reference ACTRN12613000601730; funded by Health Research Council of New Zealand.