Treatment of early‐stage mycosis fungoides: results from the PROspective Cutaneous Lymphoma International Prognostic Index (PROCLIPI) study*

Abstract

Summary Background The PROspective Cutaneous Lymphoma International Prognostic Index (PROCLIPI) study is a prospective analysis of an international database. Here we examine front‐line treatments and quality of life (QoL) in patients with newly diagnosed mycosis fungoides (MF). Objectives To identify (i) differences in first‐line approaches according to tumour‐nodes‐metastasis‐blood (TNMB) staging; (ii) parameters related to a first‐line systemic approach and (iii) response rates and QoL measures. Methods In total, 395 newly diagnosed patients with early‐stage MF (stage IA–IIA) were recruited from 41 centres in 17 countries between 1 January 2015 and 31 December 2018 following central clinicopathological review. Results The most common first‐line therapy was skin‐directed therapy (SDT) (322 cases, 81·5%), while a smaller percentage (44 cases, 11·1%) received systemic therapy. Expectant observation was used in 7·3%. In univariate analysis, the use of systemic therapy was significantly associated with higher clinical stage (IA, 6%; IB, 14%; IIA, 20%; IA–IB vs. IIA, P < 0·001), presence of plaques (T1a/T2a, 5%; T1b/T2b, 17%; P < 0·001), higher modified Severity Weighted Assessment Tool (> 10, 15%; ≤ 10, 7%; P = 0·01) and folliculotropic MF (FMF) (24% vs. 12%, P = 0·001). Multivariate analysis demonstrated significant associations with the presence of plaques (T1b/T2b vs. T1a/T2a, odds ratio 3·07) and FMF (odds ratio 2·83). The overall response rate (ORR) to first‐line SDT was 73%, while the ORR to first‐line systemic treatments was lower (57%) (P = 0·027). Health‐related QoL improved significantly both in patients with responsive disease and in those with stable disease. Conclusions Disease characteristics such as presence of plaques and FMF influence physician treatment choices, and SDT was superior to systemic therapy even in patients with such disease characteristics. Consequently, future treatment guidelines for early‐stage MF need to address these issues.

Mycosis fungoides and Sézary syndrome: Australian clinical practice statement

“…In most patients, the disease is limited to cutaneous patches or plaques, with an estimated 5‐year survival rate of >80% (see Table 2). 4 However, 20% of patients may develop cutaneous or extracutaneous tumours with an estimated 5‐year survival rate of approximately 40% (see Table 2). 5 There are three clinicopathological variants with variable prognosis listed in the EORTC‐WHO consensus statement: folliculotropic MF, granulomatous slack skin and pagetoid reticulosis.…”

Topical treatments for early-stage mycosis fungoides using Grading Recommendations Assessment, Development and Evaluation (GRADE) criteria: A systematic review

“…Treatment for early-stage, patch/plaque disease (eg, stages IA, IB, IIA) is primarily skin-directed including local radiation, phototherapy, and topical medications. 4 , 5 , 6 , 7 , 8 , 9 , 10 Within those treatment options for early-stage disease, few large, randomized, controlled studies exist, which also limits conventional systematic reviews and meta-analyses. 11 Topical therapies are the most common first-line treatments, 10 , 12 , 13 but the choice among topical steroids, retinoids, chemotherapy creams, or combination therapies may disproportionately depend on an individual dermatologist's experience and expert guidance, in addition to practical considerations such as cost and availability.…”

“… 4 , 5 , 6 , 7 , 8 , 9 , 10 Within those treatment options for early-stage disease, few large, randomized, controlled studies exist, which also limits conventional systematic reviews and meta-analyses. 11 Topical therapies are the most common first-line treatments, 10 , 12 , 13 but the choice among topical steroids, retinoids, chemotherapy creams, or combination therapies may disproportionately depend on an individual dermatologist's experience and expert guidance, in addition to practical considerations such as cost and availability. To aid dermatologists in interpreting the evidence available for topical therapies for early-stage MF, we conducted a systematic review to assess the literature on their effectiveness and adverse effects.…”

Cutaneous T‐cell lymphomas: 2021 update on diagnosis, risk‐stratification, and management

“…Moreover, an international prospective study compared skin-directed therapies (topical steroids, ultraviolet B, psoralen and ultraviolet A, topical nitrogen mustard, topical carmustine and local radiotherapy) to systemic therapy (oral retinoids, oral bexarotene, methotrexate, interferon and extracorporeal photochemotherapy) in early-stage MF. Patients receiving skin-directed therapy had a superior overall response rate 106. The limited efficacy associated with chemotherapy has been highlighted in retrospective studies in which the median time to next treatment following single or multiagent chemotherapy was ≤4 months 107,108.…”