Moxetumomab Pasudotox in Hairy Cell Leukaemia: A Profile of Its Use.

ABSTRACT

Moxetumomab pasudotox (Lumoxiti®), an anti-CD22 recombinant immunotoxin, is an important treatment option that is approved in adults with relapsed or refractory hairy cell leukaemia (HCL) who have received at least two prior lines of treatment with systemic therapies including purine nucleoside analogues. In a pivotal phase III trial, treatment with moxetumomab pasudotox resulted in approximately one third of patients achieving durable complete response lasting more than 6 months, as well as improvements in other haematological parameters and disease-related symptoms. Moxetumomab pasudotox had a generally manageable tolerability profile; the most common treatment-related adverse events (AEs) included nausea, peripheral oedema, headache and pyrexia. AEs of special interest (including haemolytic uraemic syndrome and capillary leak syndrome) were generally manageable and reversible with monitoring and supportive care.

HCL is a rare form of leukaemia (≈ 2% of all leukaemia cases) that may cause symptoms such as anaemia, easy bruising and recurrent infection due to the low production of normal blood cells. Although treatment options are available, options are limited in patients with HCL who have relapsed (disease has reappeared after remission) or are refractory (does not respond) to treatment. Moxetumomab pasudotox (Lumoxiti®) binds to a specific protein that is overexpressed on the surface of malignant B cells, and is approved to treat adults with relapsed or refractory HCL who have been treated at least twice with systemic therapies, including a purine nucleoside analogue, for HCL. In approximately one third of patients treated with moxetumomab pasudotox, no HCL cells were found in blood or bone marrow for at least 6 months; disease-related symptoms were also improved. Moxetumomab pasudotox had a generally manageable adverse event profile. While infrequent, serious adverse events such as haemolytic uraemic syndrome and capillary leak syndrome can occur, and are generally manageable and reversible with monitoring and supportive medical care (e.g. adequate oral hydration). Thus, moxetumomab pasudotox is an important treatment option in patients with relapsed or refractory HCL who have received at least two previous treatments for HCL.