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The changing therapeutic landscape, burden of disease, and unmet needs in patients with cutaneous T-cell lymphoma.

ABSTRACT: 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.

SUBMITTER: Scarisbrick JJ

PROVIDER: S-EPMC7894136 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature

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The changing therapeutic landscape, burden of disease, and unmet needs in patients with cutaneous T-cell lymphoma.

Scarisbrick Julia J JJ Bagot Martine M Ortiz-Romero Pablo L PL

British journal of haematology 20201023 4

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 random ...[more]

PMID: 33095448

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Project description:BackgroundCutaneous T-cell lymphomas (CTCL) are a group of rare non-Hodgkin lymphomas characterized by initial localization of malignant T-lymphocytes in the skin. Support and information from nurses and patient support groups have proven useful for patients with CTCL, but little is known about the educational needs of these patients.ObjectivesTo investigate the self-reported educational needs among CTCL patients using an educational needs assessment tool and to explore differences related to sex, age, disease duration, clinical stage, and education.MethodsThis observational single center study analyzed 70 patients with CTCL in routine dermatological outpatient care. The patients were asked to complete a questionnaire to capture their educational needs in regard to CTCL. The questionnaire was inspired by the educational needs assessment tool, designed and validated for patients with rheumatoid disease. The questionnaire included a general question, "In general, how much information do you want to receive about your lymphoma disease?", and five domains covering information relating to disease process (6 items), treatment (4 items), feelings (2 items), self-management of itch, sleep, and rest (2 items), and support systems (3 items). The domain scores ranged from 0 to 18 and the total score from 0 to 51, with a higher score indicating a greater need for education.ResultsWhen asked "In general, how much information do you need?", females wanted to know more compared with males (2.6 vs. 2.1, p=0.006), and patients with higher education wanted to know more than patients with lower education (2.5 vs. 2.0, p=0.025). The domains concerning treatment (80%) and disease process (75%) revealed the greatest needs for education. Patients with a disease duration <2 years reported a greater educational need for the domain support system, compared with patients with longer disease duration. Patients with lower education reported a greater educational need about feelings compared with patients with higher education.ConclusionsWe found that 65% of the CTCL patients in the cohort, particularly females, expressed a need for education, especially regarding disease process and treatment. A deeper understanding of the educational needs would enable healthcare providers to give personalized information.

Project description:Follicular lymphoma (FL) is a heterogeneous disease with varying prognosis owing to differences in clinical, laboratory, and disease parameters. Although generally considered incurable, prognosis for early- and advanced-stage disease has improved because of therapeutic advances, several of which have resulted from elucidation of the biologic and molecular basis of the disease. The choice of treatment for FL is highly dependent on patient and disease characteristics. Several tools are available for risk stratification, although limitations in their routine clinical use exist. For limited disease, treatment options include radiotherapy, rituximab monotherapy or combination regimens, and surveillance. Treatment of advanced disease is often determined by tumor burden, with surveillance or rituximab considered for low tumor burden and chemoimmunotherapy for high tumor burden disease. Treatment for relapsed or refractory disease is influenced by initial first-line therapy and the duration and quality of the response. Presently, there is no consensus for treatment of patients with early or multiply relapsed disease; however, numerous agents, combination regimens, and transplant options have demonstrated efficacy. Although the number of therapies available to treat FL has increased together with an improved understanding of the underlying biologic basis of disease, the best approach to select the most appropriate treatment strategy for an individual patient at a particular time continues to be elucidated. This review considers prognostication and the evolving treatment landscape of FL, including recent and emergent therapies as well as remaining unmet needs. IMPLICATIONS FOR PRACTICE: In follicular lymphoma, a personalized approach to management based on disease biology, patient characteristics, and other factors continues to emerge. However, application of current management requires an understanding of the available therapeutic options for first-line treatment and knowledge of current development in therapies for previously untreated and for relapsed or refractory disease. Thus, this work reviews for clinicians the contemporary data in follicular lymphoma, from advances in characterizing disease biology to current treatments and emerging novel therapies.

Dataset Information

The changing therapeutic landscape, burden of disease, and unmet needs in patients with cutaneous T-cell lymphoma.

Publications

The changing therapeutic landscape, burden of disease, and unmet needs in patients with cutaneous T-cell lymphoma.

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