Web-Based Self-Management for Patients With Lymphoma: Assessment of the Reach of Intervention of a Randomized Controlled Trial.

BACKGROUND: Randomized controlled trials (RCTs) often provide accurate estimates of the internal validity of an intervention but lack information on external validity (generalizability). We conducted an RCT on the effectiveness of a self-management intervention among patients with lymphoma in a population-based setting. OBJECTIVE: The objectives of the current study were to describe the proportion of RCT participants compared to all patients invited to participate, and compare sociodemographic and clinical characteristics of RCT participants with all respondents, all patients invited to participate, and all patients selected from the Netherlands Cancer Registry (NCR) to determine the reach of the intervention. An additional objective was to assess differences on RCT outcome variables between RCT and paper respondents. METHODS: Patients with lymphoma or chronic lymphocytic leukemia ≥18 years old at diagnosis from 13 hospitals in the Netherlands were selected from the population-based NCR, which routinely collects data on sociodemographic and clinical characteristics. Eligible patients were invited to participate in an RCT and complete a questionnaire. Web-based completion determined RCT enrollment, whereas paper respondents were followed observationally. RESULTS: A total of 1193 patients were selected from the NCR, 892 (74.77%) of whom were invited to participate in the trial by their hematologist after verifying eligibility. Among those invited, 25.4% (227/892) completed the web-based questionnaire and were enrolled in the RCT. The RCT participants were younger and there was a higher proportion of men than nonparticipants (P<.001). In addition, 25.7% (229/892) of those invited opted to participate in the paper-based observational follow-up study. Compared with paper respondents, RCT participants were younger (P<.001), with a higher proportion of men (P=.002), and had higher education levels (P=.02). RCT participants more often wanted to receive all available information on their disease (P<.001), whereas paper respondents reported higher levels of emotional distress (P=.009). CONCLUSIONS: From a population-based sample of eligible patients, the participation rate in the RCT was approximately 25%. RCT participants may not be representative of the target population because of different sociodemographic and clinical characteristics. Since RCT participants represent a minority of the target population, RCT results should be interpreted with caution as patients in the RCT may be those least in need of a self-management intervention. TRIAL REGISTRATION: Netherlands Trial Register NTR5953; https://www.trialregister.nl/trial/5790.

Health-related quality of life and persistent symptoms in relation to (R-)CHOP14, (R-)CHOP21, and other therapies among patients with diffuse large B-cell lymphoma: results of the population-based PHAROS-registry.

The increasing number of longer-living patients with diffuse large B-cell lymphoma (DLBCL) and serious side effects of treatment urged us to study the health-related quality of life (HRQoL) and persistent (treatment-related) symptoms in unselected patients after different treatment modalities and compare HRQoL of patients with a normative population. The population-based Eindhoven Cancer Registry was used to select all patients diagnosed with DLBCL from 2004 to 2010. The European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) was completed twice, with a 1-year interval. Detailed data on treatment were extracted from the Population-based HAematological Registry for Observational Studies. Two hundred fifty-six patients responded (84 %, T1). Compared to patients treated with rituximab combined with cyclophosphamide, doxorubicin, vincristine, and prednisone every 21 days ((R-)CHOP21), those who underwent (R-)CHOP14 more often reported tingling in the hands and feet (27 vs 42 %, p = 0.02) and fatigue (35 vs 46 %, p = 0.03) and reported a lower global health status/HRQoL. Mean HRQoL was statistically and clinically relevantly lower among DLBCL patients compared to a normative population (p < 0.01). Persistent tingling in hands/feet was reported more often by older patients and patients treated with (R-)CHOP14 independently of the other characteristics. Furthermore, patients who reported symptoms exhibited significantly lower HRQoL compared to patients without symptoms/worries. Patients treated with (R-)CHOP14 reported more neuropathic symptoms, more fatigue, and a lower HRQoL than patients treated with (R-)CHOP21. Alertness for persistent symptoms that occur during and after treatment of DLBCL patients is needed and may help to avoid lasting negative influence on their HRQoL.

Cost-effectiveness of rituximab as maintenance treatment for relapsed follicular lymphoma: results of a population-based study.

OBJECTIVES: On the basis of two population-based registries, our study aims to calculate the real-world cost-effectiveness of rituximab maintenance compared with observation in relapsed or refractory follicular lymphoma patients who responded to second-line chemotherapy. METHODS: Data were obtained from the EORTC20981 trial, the Netherlands Cancer Registry and two population-based registries. A Markov model was developed to calculate cost per life year gained (LYG) and quality-adjusted life years (QALYs) for three scenarios. RESULTS: Our real-world patients were (62 years) 6 to 7 years older and had higher complete response rates to second-line chemotherapy than the trial population. Differences between the real-world rituximab and observation group were observed for second-line chemotherapy and disease progression. Groups were more balanced after using propensity matching. Relying entirely on updated trial results (scenario1) in combination with local cost data resulted in ratios of €11,259 per LYG and €12,655 per QALY. For scenario2, consisting of trial efficacy and matched real-world costs, ratios of €21,202 per LYG and €23,821 per QALY were calculated. Using real-world matched evidence (scenario3) for both effectiveness and costs showed ratios of €10,591 per LYG and €11,245 per QALY. CONCLUSION: Although differences in real-world and trial population were found, using real-world data as well as results from long-term trial follow-up showed favourable ICERs for rituximab maintenance. Nevertheless, results showed that caution is required with data synthesis, interpretation and generalisability of results. As different scenarios provide answers to different questions, we recommend healthcare decision-makers to recognise the importance of calculating several cost-effectiveness scenarios.