Background:
Mantle cell lymphoma (MCL) is a heterogeneous and often aggressive malignancy characterized by a varied median overall survival ranging from 2 years to under 10 years based on risk criteria (Hoster E, et al. JCO, 2016). While response rates to front-line chemoimmunotherapy strategies can exceed 90%, patients inevitably relapse. Novel therapies, including Bruton tyrosine kinase inhibitors and immunomodulatory agents, have been approved in the relapsed/refractory settings, yet the impact of these agents on outcomes and costs in real world populations are unknown.
In an era of rapidly rising costs of therapies for hematologic malignancies, it is important to understand not only treatment patterns with the integration of novel therapies into MCL algorithms but also value, defined as incremental costs per improvement in health outcomes. Accordingly, we have designed a national prospective study of MCL patients who are initiating novel therapy.
Methods:
This study (NCT03816683) is an observational, prospective cohort study of patients with MCL treated in clinical practice. Eligibility includes persons ages 18 and older who have initiated standard or novel therapy within 3 months of the index enrollment date. 250 patients will be recruited from 50 academic (60%) and community (40%) settings in the United States. Recruitment will occur over 3 years, with a maximum follow-up of 60 months.
The primary measures of the registry are to understand treatment patterns and value with novel therapies in MCL. The secondary measures include toxicity and efficacy of these agents when administered outside of a clinical trial and their impact on health-related quality of life and healthcare resource use. The study employs a novel patient portal that will collect patient outcomes and resource utilization over time. Analyses will be conducted from the patient, physician, and payer perspectives.
Our hypothesis is that novel agents are cost-effective - as measured by costs per quality-adjusted year of life (QALY) - when used in clinical practice. Outcome measures include health state utilities (EQ-5D), (QLC-C30), survival, and healthcare resource use (HCRU), treatment modification based on adverse events, and grade 3-5 treatment-related adverse events. Uniform price weights (e.g., Medicare reimbursements) will be applied to HCRU to estimate costs. Incremental cost-utility estimates will be calculated both for the within-study period and over a lifetime, the latter using modeling.
Conclusions:
As the first observational study specifically designed to evaluate the clinical and economic outcomes of novel therapies for MCL in clinical practice, the findings will have high relevance for clinicians, payers, and patients.
Ujjani:AstraZeneca: Consultancy; Genentech: Consultancy; Rigel: Consultancy; Gilead: Consultancy; Abbvie: Research Funding; Pharmacyclics: Research Funding. Mato:Celgene: Consultancy; Johnson & Johnson: Consultancy, Research Funding; AstraZeneca: Consultancy, Research Funding; AbbVie: Consultancy, Research Funding; Sunesis: Consultancy, Research Funding; TG Therapeutics: Consultancy, Other: DSMB member , Research Funding; LOXO: Consultancy, Research Funding; DTRM Biopharma: Research Funding; Genentech: Consultancy, Research Funding; Pharmacyclics: Consultancy, Research Funding; Gilead: Research Funding; Acerta: Consultancy; Janssen: Consultancy. Cohen:Bristol-Meyers Squibb Company: Research Funding; Takeda Pharmaceuticals North America, Inc.: Research Funding; Gilead/Kite: Consultancy; LAM Therapeutics: Research Funding; UNUM: Research Funding; Hutchison: Research Funding; Astra Zeneca: Research Funding; Lymphoma Research Foundation: Research Funding; ASH: Research Funding; Genentech, Inc.: Consultancy, Research Funding; Janssen Pharmaceuticals: Consultancy; Seattle Genetics, Inc.: Consultancy, Research Funding. Svoboda:Kite: Consultancy; Pharmacyclics: Consultancy, Research Funding; AstraZeneca: Consultancy; Celgene: Research Funding; Kyowa: Consultancy; Seattle Genetics: Consultancy, Research Funding; BMS: Consultancy, Research Funding; Merck: Research Funding; Incyte: Research Funding. Andorsky:AstraZeneca: Consultancy; Genetech: Research Funding; Gilead: Research Funding; CTI: Research Funding; Celgene: Research Funding. Goy:Takeda: Other: Grants outside of the submitted work; Celgene: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; University of Nebraska: Research Funding; Hakensackumc: Research Funding; Acerta: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Grants outside of the submitted work, Research Funding; Hackensack University Medical Center, RCCA: Employment; Kite, a Gilead Company: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Grants outside of the submitted work; Pharmacyclics/Janssen: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Grants outside of the submitted work, Research Funding; Astrazenca: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Genentech: Other: Grants outside of the submitted work, Research Funding; COTA: Equity Ownership, Membership on an entity's Board of Directors or advisory committees, Other: leadership role for profit healthcare company. Wang:Juno Therapeutics: Research Funding; MoreHealth: Consultancy, Equity Ownership; Guidepoint Global: Consultancy; Kite Pharma: Consultancy, Research Funding; Loxo Oncology: Research Funding; Celgene: Honoraria, Research Funding; BioInvent: Consultancy, Research Funding; VelosBio: Research Funding; Aviara: Research Funding; Dava Oncology: Honoraria; Acerta Pharma: Consultancy, Research Funding; Janssen: Consultancy, Honoraria, Research Funding, Speakers Bureau; Pharmacyclics: Honoraria, Research Funding; AstraZeneca: Consultancy, Honoraria, Research Funding, Speakers Bureau. Kumar:Pharmacyclics: Research Funding; Celgene: Research Funding, Speakers Bureau; Astra Zeneca: Other: Steering Committee for MCL Registry ; Adaptive Biotechnologies: Research Funding; Seattle Genetics: Research Funding; Abbvie Pharmaceuticals: Research Funding. Dawson:AstraZeneca: Employment, Equity Ownership. Elesinmogun:AstraZeneca: Employment, Equity Ownership. Kabadi:AstraZeneca: Employment, Equity Ownership. Seal:AstraZeneca: Employment, Equity Ownership. Pagel:Pharmacyclics: Consultancy; AstraZeneca: Consultancy; Gilead Sciences: Consultancy. Ramsey:ASCO Quality: Other: $1500 honorarium that he will receive as a speaker at.
Author notes
Asterisk with author names denotes non-ASH members.
