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: Hairy cell leukemia is a rare indolent B-cell malignancy, characterized by pancytopenia, recurrent infections and splenomegaly. After initial therapy with purine nucleoside analogs, up to 50% of patients relapse after several years of remission. The number of relapsed patients is increasing and, until recently, there was no approved therapy with durable responses for hairy cell leukemia patients in the relapsed setting, thus the need for new non-chemotherapy approach with significant efficacy and less myelosuppression. : Moxetumomab pasudotox is a recombinant immunotoxin containing an Fv fragment of an anti-CD22 monoclonal antibody and truncated (PE38). The authors reviewed pre-clinical and clinical studies that led to the FDA approval of the drug in patients with relapsed and/or refractory hairy cell leukemia, who received at least two prior therapies, including at least one purine nucleoside analog. : Moxetumomab pasudotox demonstrated a durable complete remission rate of 30% in heavily pretreated patients with hairy cell leukemia, and MRD eradication in 85% of responding patients. Moxetumomab pasudotox got a global FDA approval in September 2018. The US prescribing information carries boxed warnings regarding the risk of capillary leak syndrome and hemolytic uremic syndrome. Long-term follow-up of the pivotal study is ongoing (NCT01829711).
This article was published in the following journal.
Name: Expert review of hematology
Moxetumomab pasudotox is a promising new therapy for the treatment of patients with relapsed/refractory hairy cell leukemia (R/R HCL), but practical guidance relating to its administration is limited.
Hairy cell leukemia (HCL) is a rare mature B cell leukemia. Purine analogs are the mainstay of treatment of HCL, but relapse after purine analog therapy is common. Outcomes of treatment of relapsed/re...
The majority of patients with acute myeloid leukemia (AML) die from disease recurrence and historically, treatment options in both the relapsed and refractory settings of this disease have been limite...
The article Moxetumomab Pasudotox: First Global Approval, written by Sohita Dhillon, was originally published Online First without open access.
Patients with relapsed or refractory acute lymphoblastic leukemia (R/R ALL) have dismal outcomes, with survival of less than 6 months, and treatment options in the salvage setting have been limited to...
Early Access Programme to provide treatment access to moxetumomab pasudotox for eligible patients with relapsed/refractory hairy cell leukemia
The goal of this clinical research study is to find the highest tolerable dose of moxetumomab pasudotox that can be given to patients with relapsed and/or refractory ALL.
Background: Hairy cell leukemia (HCL) is a rare, slow-growing blood cancer in which the bone marrow makes too many of certain white blood cells. The antibody Rituximab binds to a protein ...
This phase II trial studies how well ibrutinib works in treating patients with relapsed hairy cell leukemia. Ibrutinib may stop the growth of cancer cells by blocking some of the enzymes n...
Hairy-cell leukemia is a rare and indolent lymphoid disorder, representing 2% of all cases of lymphoid leukemias. Treatment of hairy-cell leukemia relies mainly on the purine analogs, clad...
A neoplastic disease of the lymphoreticular cells which is considered to be a rare type of chronic leukemia; it is characterized by an insidious onset, splenomegaly, anemia, granulocytopenia, thrombocytopenia, little or no lymphadenopathy, and the presence of "hairy" or "flagellated" cells in the blood and bone marrow.
An antineoplastic agent used in the treatment of lymphoproliferative diseases including hairy-cell leukemia.
A potent inhibitor of ADENOSINE DEAMINASE. The drug induces APOPTOSIS of LYMPHOCYTES, and is used in the treatment of many lymphoproliferative malignancies, particularly HAIRY CELL LEUKEMIA. It is also synergistic with some other antineoplastic agents and has immunosuppressive activity.
T Lymphocytes with limited diversity of receptors (e.g., ALPHA E INTEGRINS) in the epidermis of the skin and the mucosal linings. They recognize common microbes via T-CELL RECEPTORS and PATHOGEN-ASSOCIATED MOLECULAR PATTERN MOLECULES and function as effector cells for INNATE IMMUNITY. Activation of intraepithelial lymphocytes is a marker for various gastrointestinal diseases (e.g., CELIAC DISEASE; HAIRY CELL LEUKEMIA; and ENTEROPATHY-ASSOCIATED T-CELL LYMPHOMA).
The phase of chronic myeloid leukemia following the chronic phase (LEUKEMIA, MYELOID, CHRONIC-PHASE), where there are increased systemic symptoms, worsening cytopenias, and refractory LEUKOCYTOSIS.