Clinical end points and response criteria in mycosis fungoides and Sézary syndrome: a consensus statement of the International Society for Cutaneous Lymphomas, the United States Cutaneous Lymphoma Consortium, and the Cutaneous Lymphoma Task Force of the European Organisation for Research and Treatment of Cancer

Abstract

Mycosis fungoides (MF) and Sézary syndrome (SS), the major forms of cutaneous T-cell lymphoma, have unique characteristics that distinguish them from other types of non-Hodgkin's lymphomas. Clinical trials in MF/SS have suffered from a lack of standardization in evaluation, staging, assessment, end points, and response criteria. Recently defined criteria for the diagnosis of early MF, guidelines for initial evaluation, and revised staging and classification criteria for MF and SS now offer the potential for uniform staging of patients enrolled in clinical trials for MF/SS. This article presents consensus recommendations for the general conduct of clinical trials of patients with MF/SS as well as methods for standardized assessment of potential disease manifestations in skin, lymph nodes, blood, and visceral organs, and definition of end points and response criteria. These guidelines should facilitate collaboration among investigators and collation of data from sponsor-generated or investigator-initiated clinical trials involving patients with MF or SS.

Conflict of interest statement

Authors' disclosures of potential conflicts of interest and author contributions are found at the end of this article.

Figures

Fig A1.

An example of how one determines the modified Severity Weighted Assessment Tool (mSWAT) score is given here for a patient with T3 stage skin disease who has predominantly patches and plaques. The lesions were catalogued in person and recorded. Although standardized photographs are generally representative of the extent of lesions, they are an inadequate method of making a mSWAT determination. Photographs do not capture subtle erythema or elevation or induration of lesions, thus making the designation between patch and plaque, and plaque and tumor, difficult, and they do not fully capture the involvement in the intertriginous areas, groin, scalp, and soles of feet unless far more extensive photographs than usual are taken at study visits. However, the photographs presented here will give the reader an idea of how to calculate an mSWAT score and why the ideal in a clinical trial setting is for one person to do the assessments at each visit. Photographs of this patient's face and groin are purposefully not shown. This patient's anterior and posterior trunk are outlined in black in panels (A) through (D). In these areas, because of truncal obesity, it is useful to determine lesional BSA by consideration of the % of the regional BSA involved (anterior and posterior trunk are each estimated at 13% BSA) by each lesion subtype as well as by using the patient's palmar hand and fingers to approximate 1% BSA to avoid overestimation of involvement in these areas. The % BSA covered by patches (enclosed within blue broken lines) on the anterior trunk is approximately 3.2% and that of plaques (enclosed within red lines) is approximately 0.7% BSA. The % BSA covered by patches on the posterior trunk is approximately 2%, and the % BSA covered by plaques is approximately 0.7%. This patient has ulcerated plaques, which the International Society for Cutaneous Lymphomas/European Organisation for Research and Treatment of Cancer has previously agreed should be counted as plaques and not tumors. If one uses the same process for the other areas of the body, shown in panels (E) through (O), then the total BSA covered by patches is approximately 9%, the total BSA covered by plaques is approximately 2.9%, and the total BSA covered by tumors (enclosed within white lines) is 0.1% BSA. Multiplying these numbers by the weighting factors of 1 for patch, 2 for plaque, and 4 for tumor, the final mSWAT score is 15.2. To determine maximum response or loss of response/progressive disease in the skin, the mSWAT at a particular time point would be compared with that of baseline or time of maximal response respectively.