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Cutaneous T-cell lymphomas (CTCLs) have a chronic, relapsing course, and the most common subtypes are mycosis fungoides and Sézary syndrome. The disease causes visible skin alterations and can also cause alopecia, pruritus and pain, all of which can impact patients' health-related quality of life (HRQoL). The goal of treatment is to reduce symptoms and prevent disease progression. However, treatment recommendations are often based on low levels of evidence due to the lack of well-designed randomised clinical trials and treatment guidelines, and approved drugs vary considerably across different countries and regions. Currently, available treatments rarely lead to durable remissions and eventually become less effective, meaning patients often require multiple therapy changes. Skin-directed therapies (SDTs) are first-line treatments for early-stage CTCL, whereas systemic therapies may be needed for early-stage disease that does not respond to SDT or for advanced-stage disease. However, patients can experience significant side-effects with these treatments or may be unable to tolerate them. Hence, there is an unmet need for effective therapies with good safety profiles for the treatment of early- and late-stage CTCL. Here, we review current treatment guidelines, investigational and approved treatments, the impact of CTCL on patients' HRQoL, and the treatment of pruritus., (© 2020 The Authors. British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.)

Purpose: To evaluate health care utilization, treatment patterns and costs among patients with mycosis fungoides-cutaneous T-cell lymphoma (MF-CTCL)., Methods: This retrospective cohort study queried the HealthCore Integrated Research Database to identify patients ≥18 years with ≥2 diagnoses of MF-CTCL (ICD-9-CM code 202.1x, 202.2x) between 07 January 2006 and 07 January 2013. Index date was defined as first MF-CTCL diagnosis. Patients were continuously enrolled ≥6 months before and ≥12 months after index date. Severe MF-CTCL was identified via systemic therapy use postindex. Generalized linear model (GLM) was used to estimate the relationship between MF-CTCL severity and healthcare costs controlling for selected factors., Results: A total of 1981 MF-CTCL patients were evaluated: 493 (24.9%) severe and 1488 (75.1%) with mild to moderate disease. GLM analysis indicated severe MF-CTCL patients incurred higher all-cause healthcare total costs compared to patients with mild-to-moderate MF-CTCL (coefficient estimate: 4.19, p < .0001). About 51% of patients did not receive any MF-CTCL-specific treatment within 60 days after MF-CTCL diagnosis., Conclusions: MF-CTCL severity was associated with greater healthcare resource utilization and costs. These findings suggest that about half of MF-CTCL patients do not receive MF-CTCL-specific treatment within 60 days following initial diagnosis. Future studies are needed to understand reasons for delayed treatment initiation.

Purpose: To conduct an exploratory cost-effectiveness analysis of systemic treatment options for more advanced cutaneous T-cell lymphoma (CTCL)., Methods: A cost-effectiveness model compared systemic bexarotene, denileukin diftitox, interferon-α, methotrexate, pralatrexate, romidepsin, vorinostat, and extracorporeal photopheresis (ECP) treatment of CTCL. Treatment effectiveness data were extracted from published studies and/or US product labeling. Overall response, the primary effectiveness measure, was defined as the proportion of patients achieving complete or partial response. Costs were based on wholesale acquisition cost (medications) and Medicare reimbursement rates (ECP, medication administration, adverse drug effect treatment). The perspective of the study was from that of a payer., Results: Methotrexate was the lowest cost option [mean $436; standard deviation (SD) $284], followed by interferon-α (mean $32,174; SD $27,582), denileukin difitox (mean $40,107; SD $18,598), and ECP (mean $40,985; SD $45,633). Other treatments had costs greater than $50,000, ranging from vorinostat ($65,958; SD $40,637) to bexarotene ($239,424; SD $178,881). The incremental cost-effectiveness ratio per successfully treated patient was $396,725 (interferon) and $213,416 (ECP). Denileukin diftitox, romidepsin, and vorinostat were less effective and cost more than methotrexate., Conclusion: Methotrexate is the most cost-effective option for CTCL; however, its low cost is offset by its limited effectiveness in advanced stages of CTCL. ECP and interferon appear the next most cost-effective therapies.

It is apparent that multiple end points are required to adequately describe the response to therapy for CTCL. Investigators may differ as to which end points are best suited for their data. In general, multiple end points provide a better overall view of response to treatment as compared with a single end point. Regardless, measures of response and end points should be clearly defined and the methods of calculating end points clearly stated.