EJHaem最新文献

Background

Chronic lymphocytic leukemia (CLL) treatment choice remains a challenge in the era of molecular biology and targeted therapy.

Methods

We conducted a bicentric retrospective analysis of the impact of NOTCH1 mutation according to the treatment of CLL patients in real life.

Results

A total of 45 patients with NOTCH1 mutation have been reported, including 15 patients treated with FCR BR or DRC regimen and 18 patients with a BTK inhibitor. NOTCH1 mutation is the most frequently occurring molecular abnormality in CLL and is closely associated with poor prognosis but is not used in treatment guidelines unlike TP53 and IGHV. In our study, progression-free survival was significantly longer in CLL patients with NOTCH1 mutation treated by BTK inhibitors compared to immunochemotherapy.

Conclusion

Routine screening for NOTCH1 mutations could identify patients who may benefit from BTKi treatment. However, the impact of NOTCH1 mutations on combination therapies, such as obinutuzumab-venetoclax or venetoclax-BTKi, is yet to be determined.

The authors have confirmed clinical trial registration is not needed for this submission

Background

Venous thromboembolism (VTE) is a serious complication in cancer patients, with malignancy increasing the risk significantly. Direct oral anticoagulants (DOACs) have emerged as a convenient alternative to traditional therapies, though optimal dosing remains uncertain.

Methods

We performed a systematic review and meta-analysis on three studies. A comprehensive literature search was performed on PubMed, Embase, the Cochrane Library, and ScienceDirect till April 2025. Analysis was carried out on RevMan 5.4. The risk of bias was assessed via RoB 2.0.

Results

A total of three studies with 2416 participants were identified, including 1495 patients in the reduced-dose group and 1232 patients in the full-dose group. No significant difference was observed in recurrent VTE (OR 0.70, 95% CI 0.45–1.09, p = 0.11) or recurrent symptomatic VTE (OR 0.96, 95% CI 0.50–1.84, p = 0.91). However, reduced-dose DOACs were associated with a significantly lower incidence of incidental VTE (OR 0.31, 95% CI 0.14–0.69, p = 0.004). The reduced-dose group also had a lower incidence of CRNMB plus major bleeding (OR 0.69, 95% CI 0.55–0.88, p = 0.002).

Conclusions

In terms of venous thromboembolism, bleeding events, and all-cause mortality, reduced-dose DOACs demonstrated a safety profile that was either superior or comparable to that of full-dose DOACs.

Trial Registration

The authors have confirmed clinical trial registration is not needed for this submission

Introduction

A novel therapy for heavily pretreated triple-class–exposed multiple myeloma (TCE MM) is B-cell maturation antigen (BCMA)-targeted immunotherapy. While the number of TCE+BCMA-exposed patients is growing, real-world data for this group are limited.

Methods

We present real-world data from patients with TCE+BCMA-exposed MM who initiated a subsequent line of therapy (LOT) using a US-based claims database, Komodo's Healthcare Map.

Results

We identified 656 TCE+BCMA-exposed patients; mean age was 66.5 years. Time from MM diagnosis to index was 5.4 years; mean number of prior LOTs was 5.9. The most prevalent prior therapy received within each drug class was daratumumab (98.5%), pomalidomide (86.0%), carfilzomib (85.8%) and belantamab mafodotin (74.5%). A total of 137 different subsequent treatment regimens were observed following TCE+BCMA exposure; the most common regimen was teclistamab (10.4%). The top three targeted agents within the subsequent regimen were carfilzomib (20.2%), pomalidomide (20.1%) and bortezomib (16.6%). Among this TCE+BCMA-exposed population who received subsequent treatment, the median time to next treatment or death was 6.8 (95% CI, 6.1–7.5) months; time to treatment discontinuation or death was 3.5 (95% CI, 3.2–3.7) months.

Conclusion

This first real-world analysis of patients with heavily pretreated TCE+BCMA-exposed MM shows poor clinical outcomes, frequent therapy retreatment and no standard-of-care, highlighting the need for novel treatments.

Clinical Trial Registration

The authors have confirmed clinical trial registration is not needed for this submission.

Background

Some injectable medicines introduced recently allow patients with multiple myeloma (MM) to receive their chemotherapy at home. This study aimed at describing adult patients with MM receiving injectable chemotherapy via hospital-at-home (HAH) services and outpatient hospital units (OHUs) in metropolitan France in 2019 and 2020, analyzing the factors influencing HAH use, and evaluating the geographic variations and the evolution over time of HAH use by these patients during the study period.

Methods

Real-world data from the French Hospital Discharge Database (PMSI) were analyzed.

Results

In total, 2169/9278 patients (23.4%) received at least one HAH chemotherapy injection. These patients were diagnosed more recently (mean ± standard deviation = 25.1 ± 19.6 vs. 31.6 ± 21.8 months), and lived in larger and wealthier cities (59,000 vs. 41,000 inhabitants; €23,300 ± €5300 vs. €21,700 ± €4100) and closer to their follow-up hospital (18.7 ± 18.4 vs. 31.3 ± 31.2 km) than patients exclusively treated in OHUs (p < 0.001). Receiving bortezomib and carfilzomib, and the first chemotherapy dose in 2020, were the most significant factors associated with HAH use (odds ratio [95% confidence interval]: 6.12 [5.40–6.96], 2.01 [1.69-2.39], and 1.81 [1.57–2.09], respectively, p < 0.001). HAH use increased between 2019 and 2020 (patients, +23%; administrative departments, +25%), likely related to the COVID-19 pandemic. However, HAH use remained limited overall and exhibited inter-regional variability. Infection-related hospitalizations remained stable.

Conclusions

Receiving chemotherapy injections at home is feasible and safe, but further development and equitable access are essential to enhance patients’ quality of life and reduce costs.

Introduction

We previously reported that sodium-glucose co-transporter 2 (SGLT-2) was ectopically overexpressed in adult T-cell leukemia (ATL) cells notably in aggressive type but in indolent type, and widely-used anti-diabetic SGLT-2 inhibitors (SGLT-2i) considerably attenuated proliferation of leukemic cells.

Methods

We performed retrospective analyses for 10 years to see whether SGLT-2i would prevent aggressive transformation in patients with indolent type ATL accompanied by diabetes. Nucleosome occupancy in the promotor region of the SGLT-2 gene was also assessed to explore the possible involvement of epigenetic modification in such an ectopic overexpression.

Results

In patients of indolent ATL with diabetes, the cumulative progression rate in the non-SGLT-2i-treated group was 71%, while no patients developed aggressive transformation in the SGLT-2i treated group. ATL cells showed an apparent trend to decrease nucleosome occupancy in the promotor region of the SGLT-2 gene.

Conclusion

Our data suggest that SGLT-2i is advantageous for preventing aggravative transformation in indolent ATL.

Trial Registration

Authors confirmed that clinical trial registration was not requested for the present study and this manuscript.

Introduction

Immune thrombocytopenia (ITP) is characterised by a low platelet count and increased risk of bleeding. Recent research has also proposed that having ITP increases thrombosis risk. Moreover, certain ITP treatments have been associated with an increased risk of thrombosis. This Delphi study aims to assess haematologist opinion regarding aspects of optimise thrombotic risk management in primary ITP in the UK.

Methods

The methodology employed a modified Delphi process with two rounds of evaluation from a panel of experts. A literature review on the topic of primary ITP generated input to a steering group of three experts from the UK attended a virtual meeting in May 2024. During this meeting, and guided by an independent facilitator, the group identified five main domains. From these, 42 statements were agreed and developed into an online survey for testing with a wider panel of peers.

Results

Overall, 33 statements achieved consensus agreement, and one statement did not achieve consensus. Eight scenario statements were included to identify preferable treatment options among healthcare professionals.

Conclusion

Based on the agreement levels achieved, the steering group formulated a set of recommendations to optimise the management of thrombotic risk in patients with primary ITP in the UK.

Trial Registration

The authors have confirmed clinical trial registration is not needed for this submission.

Background

Healthcare is shifting from a disease-centered to patient-centered approach, and aspects of health such as quality of life (QoL) are becoming increasingly relevant. “E-Res Salud” for hematological malignancies is a value-based healthcare program aiming to improve patient experience and outcomes. The program collects e-PROMs via automatically deployed, validated questionnaires over a mobile application.

Methods

A multicenter prospective observational cohort study including 243 patients with Hodgkin and non-Hodgkin lymphoma receiving outpatient intravenous immunochemotherapy at four teaching hospitals in Madrid, Spain, of whom 121 participated in the “E-Res Salud” program.

Results

We found that adverse event reporting differs significantly between patients and healthcare professionals. Participants showed significantly lower rates of emergency department visits (37.2% vs. 56.6%; p < 0.01) and unplanned hospital admissions (21.5% vs. 32.8%; p < 0.05), as well as significantly higher rates of treatment completion and overall survival (88.4% vs. 79.5% after 18 months of follow-up; stratified hazard ratio, 2.30; 95% CI 1.25–4.22; p = 0.007).

Conclusions

An e-PROMS program for patients with lymphoma is associated with lower use of healthcare and improved clinical outcomes. Patients and hematologists report adverse events differently, demonstrating the importance of patient-reported outcome measurement to improve symptom management in clinical practice.

Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission

Introduction

With the purpose of identifying unmet needs of patients with acute leukemia, survivors were identified from the Swedish National Acute Leukemia registries.

Methods

At six months and at two years from diagnosis, patients were requested by mail to report outcome with focus on depression, sick leave, and sexual dysfunction.

Results

Of 910 patients alive at 6 months, 474 (52%) participated, and of 331 alive at two years, 250 (76%) participated. At two years, the majority ≤65 years, 52%, had returned to work >30 h/week, and 40% were still on medical leave. Economic hardship was common, especially in the latter group. Impaired sexual function, impact on sexual desire, as well as anxiety about infections, were frequently reported. Depression with a PHQ-8 score ≥10 was more prevalent in patients ≤65 years than in older patients. In younger patients, a PHQ-8 score ≥10 was associated with sexual dysfunction, economic hardship, single living, and anxiety of infections, whereas in patients >65 years, single living and economic hardship remained significant. No impact of the Covid-19 pandemic on depression was found.

Conclusion

The use of depression screening instruments and awareness of sexual dysfunction could be of importance in the routine care of acute leukemia patients two years after diagnosis.

Trial Registration

The authors have confirmed clinical trial registration is not needed for this submission.