Emerging treatments
Denileukin diftitox
Denileukin diftitox is a recombinant fusion protein composed of diphtheria toxin fragments and human interleukin (IL)-2, which targets the IL-2 receptor on malignant T cells and regulatory T cells. It has been approved by the Food and Drug Administration (FDA) for the treatment of patients with relapsed or refractory stages I-III cutaneous T-cell lymphomas (CTCL) who have undergone at least one prior line of systemic therapy. Denileukin diftitox was first approved for the treatment of CTCL in 2008, but was discontinued in 2014 due to production issues related to theEscherichia coli expression system and purification challenges. The newly approved formulation has improved purity and bioactivity compared to the original product. Denileukin diftitox is recommended by the NCCN as a preferred treatment option for patients with stage II B MF, tumour stage disease.[32] However, this treatment will not be available in the US until 2025. Denileukin diftitox is not currently approved in Europe. One phase III multicentre, open label single arm trial demonstrated a clinically meaningful rapid, durable, and deep tumour response for heavily pre-treated patients with relapsed/refractory CTCL who received denileukin diftitox.[113]
KT-333
KT-333, a first-in-class degrader of the transcriptional regulator STAT3, has been granted orphan drug designation for the treatment of CTCL by the FDA. A phase Ib trial is ongoing.[114]
Topical therapies
A number of topical treatments have been reported as effective treatment for mycosis fungoides in case reports or small series (off-label use) including fluorouracil and tacrolimus. Randomised trials have not been done. However, these therapies may be considered for localised or refractory disease when skin-directed therapy is still considered appropriate.
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