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Background Non-Hodgkin lymphoma (NHL) constitutes a biologically and clinically heterogeneous group of lymphoid malignancies, with varying prognoses and treatment aims between indolent and aggressive subtypes. While survival outcomes remain key efficacy measures, they insufficiently capture the impact of disease and treatment on daily physical and psychosocial functioning, as well as quality of life (QoL). Objectives To characterize the use and reporting of patient-reported outcomes (PROs) in adult NHL randomized controlled trials (RCTs), including the trajectory of PRO dissemination throughout the trial reporting process. Methods MEDLINE, CENTRAL, ClinicalTrials.gov, and WHO ICTRP were systematically searched from 1 January 2017 to 19 January 2023 for NHL RCTs irrespective of subtype, with databases being re-searched until 4 February 2025. Cross-sectional and longitudinal descriptive analyses were conducted to investigate PRO use and reporting practices. Findings 549 completed and ongoing RCTs met the eligibility criteria, of which 176 trials (32.1%) included a reference to PROs, and 27 (4.9%) used a PRO as a primary outcome. Overall, 95 PRO measures were identified across trials, with EORTC QLQ-C30, FACT-Lymphoma, and EQ-5D being referenced most frequently. Among trials with published results that pre-specified PROs, 25.0% entirely and 10.5% partly omitted PRO findings from their main full-texts, without addressing this shortcoming in subsequent publications. Conclusion PROs are underused and underreported in RCTs in NHL. Standardized PRO evaluation methodology and guidance for the selection of fit-for-purpose PRO measures are needed, along with clear positioning of PROs alongside survival outcomes in clinical trial design and interpretation.

Background: Patient-reported outcomes (PROs) are becoming increasingly important in supporting clinical outcomes in clinical trials. In multiple myeloma (MM), PRO measurement is useful to reveal how treatment affects physical, psychosocial, and functional behaviour as well as symptoms and treatment-related adverse events to evaluate the benefit-risk ratio of a particular drug or drug combination. We report the types of PRO instruments used in MM, the frequency in which they are utilised in randomised controlled trials (RCTs), and the consistency of their reporting. Methods: The European Hematology Association (EHA) supports the development of guidelines for the use of PROs in adult patients with haematological malignancies. The first step is the present systematic review of the literature. MEDLINE and CENTRAL were searched for RCTs in MM between 2015 and 2020. Study design, characteristics of MM and its treatment, the primary outcomes, and the types of PRO instrument(s) were extracted using a predefined template. Additionally, in a stepwise approach, it was assessed whether the identified instruments had been validated for multiple myeloma patients, patients with haematological malignancies, or cancer patients. Results: Following screening for RCTs, 283 studies were included for review from 10,707 records retrieved, and 118 of these planned the use of PRO measures. Thirty-eight PRO instruments were reported. The most frequently used instrument (92 studies) was the EORTC QLQ-30. The EORTC-MY20 MM-specific questionnaire was the second most frequently used (50 studies), together with the EQ-5D (50 studies). Only 19 PRO instruments reported were consistent with the trial registry. Furthermore, in 58 publications, the information on PRO instruments differed between the publication and the trial registry. Further, information on PRO in HTA reports was available for 26 studies, of which 18 reports were consistent with the trial registries. Out of the 38 instruments used, six had been validated for patients with multiple myeloma (the most frequently used), six for patients with haematological malignancies, and 10 for cancer patients in general. Conclusions: The findings indicate that the measurement of PROs in RCTs for MM is underutilised, underreported, and often inconsistent. Guidelines for the appropriate use of PROs in MM are needed to ensure standardisation in selection and reporting. Furthermore, not all PRO instruments identified have been validated for myeloma patients or patients with haematological malignancies. Thus, guidelines for the appropriate use and reporting of PROs are needed in MM to ensure standardisation in the selection and reporting of PROs.

In the era of personalized medicine there is an increasing need for the assessment of patient-reported outcomes (PROs) to become a standard of patient care. Patient-reported outcome measures (PROM) are important in assessing significant and meaningful changes as a result of an intervention based on a patient’s own perspective. It is well established that active multiple myeloma (MM) can be characterized by a high burden of disease and treatment-related symptoms, with considerable worsening of quality of life (QoL). In general, and over the past decade, the focus has shifted to obtaining the most durable remissions with the best QoL as primary goals for MM treatment. Patients place considerable value on their QoL and communicating about QoL data prior to treatment decisions allows them to make informed treatment choices. Consequently, optimization of QoL of patients with MM is an important therapeutic goal and the incorporation of PROs into clinical trials has the potential of improving treatment outcomes. In this regard, guidance for the use and reporting of PROMs in MM in clinical trials is warranted. Under the auspices of the European Hematology Association, evidence-based guidelines for the use and reporting of PROs in patients with MM have been developed according to the EHA’s core Guidelines Development Methodology. This document provides general considerations for the choice of PROMs in MM clinical trials as well as a series of recommendations covering a selection of PROMs in MM clinical trials; the mode of administration; timing of assessments; strategies to minimize missing data; sample size calculation; reporting of results; and interpretation of results.

The objective of this scoping review is to summarize the current use of pharmacokinetics for tailoring prophylaxis in hemophilia patients switching between clotting factor products. Patients with hemophilia may require switching of clotting factor concentrates due to a variety of factors, but there have been perceived risks associated with switching, such as inhibitor development or suboptimal protection due to inadequate dosing while titrating treatment. Studies that look at patients switching from one clotting factor concentrate to another are categorized in terms of their primary and/or secondary objectives, notably biosimilarity and comparative pharmacokinetic studies and inhibitor development studies. Research on how best to switch concentrates with respect to dosing regimen are lacking, and currently a trial‐and‐error approach is used for dosing the new factor concentrate. In the future, studies looking at the predictability of pharmacokinetics (PK) of a new factor concentrate based on individual PK knowledge of the original factor concentrate may offer clinical benefit by providing a safer switching approach and protocol.

Estonia regained independence in 1991 after five decades of occupation by the Soviet Union. The present population-based survey was carried out over five consecutive 5-year study periods (1982–2006) on the incidence and survival of de novo acute leukemia patients aged ≥65 years at diagnosis in Estonia and in a well-defined area in western Sweden. During the study period of retrospective work (1982–1996), the first 10 years were carried out while Estonia was still under the mentorship of the Soviet Union. Over these years, Estonian hematologists did not have access to therapeutic measures readily available to Swedish hematologists, and the results for survival for western Swedish patients with acute myeloid leukemia (AML) far exceeded those of their Estonian counterparts. However, the results for acute lymphoblastic leukemia were equally dismal in the two countries. Subsequent prospective population-based studies were carried out during the years 1997–2006. A gradual improvement as to long-term relative survival of the Estonian AML patients was observed. When studying 2002–2006, no difference as regards relative survival at 5 years was anymore present between the two countries. Over the first 20 years of our population-based studies, it was repeatedly observed that the age-standardized incidence rate particularly for de novo AML was considerably higher for the western Swedish as compared to the Estonian cohorts. During the last 5-year study period (2002–2006), no such difference between the two countries was present, indicating that some true changes in the reporting procedure in Estonia had occurred.

Estonia regained independence in 1991 after five decades of occupation by the Soviet Union. The present population-based survey was carried out over five consecutive 5-year study periods (1982â[euro]\"2006) on the incidence and survival of de novo acute leukemia patients aged â[per thousand]¥65Â years at diagnosis in Estonia and in a well-defined area in western Sweden. During the study period of retrospective work (1982â[euro]\"1996), the first 10Â years were carried out while Estonia was still under the mentorship of the Soviet Union. Over these years, Estonian hematologists did not have access to therapeutic measures readily available to Swedish hematologists, and the results for survival for western Swedish patients with acute myeloid leukemia (AML) far exceeded those of their Estonian counterparts. However, the results for acute lymphoblastic leukemia were equally dismal in the two countries. Subsequent prospective population-based studies were carried out during the years 1997â[euro]\"2006. A gradual improvement as to long-term relative survival of the Estonian AML patients was observed. When studying 2002â[euro]\"2006, no difference as regards relative survival at 5Â years was anymore present between the two countries. Over the first 20Â years of our population-based studies, it was repeatedly observed that the age-standardized incidence rate particularly for de novo AML was considerably higher for the western Swedish as compared to the Estonian cohorts. During the last 5-year study period (2002â[euro]\"2006), no such difference between the two countries was present, indicating that some true changes in the reporting procedure in Estonia had occurred.[PUBLICATION ABSTRACT]