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|Ref Type||Journal Article|
|Authors||Kreitman RJ, Dearden C, Zinzani PL, Delgado J, Karlin L, Robak T, Gladstone DE, le Coutre P, Dietrich S, Gotic M, Larratt L, Offner F, Schiller G, Swords R, Bacon L, Bocchia M, Bouabdallah K, Breems DA, Cortelezzi A, Dinner S, Doubek M, Gjertsen BT, Gobbi M, Hellmann A, Lepretre S, Maloisel F, Ravandi F, Rousselot P, Rummel M, Siddiqi T, Tadmor T, Troussard X, Yi CA, Saglio G, Roboz GJ, Balic K, Standifer N, He P, Marshall S, Wilson W, Pastan I, Yao NS, Giles F|
|Title||Moxetumomab pasudotox in relapsed/refractory hairy cell leukemia.|
|Abstract Text||This is a pivotal, multicenter, open-label study of moxetumomab pasudotox, a recombinant CD22-targeting immunotoxin, in hairy cell leukemia (HCL), a rare B cell malignancy with high CD22 expression. The study enrolled patients with relapsed/refractory HCL who had ≥2 prior systemic therapies, including ≥1 purine nucleoside analog. Patients received moxetumomab pasudotox 40 µg/kg intravenously on days 1, 3, and 5 every 28 days for ≤6 cycles. Blinded independent central review determined disease response and minimal residual disease (MRD) status. Among 80 patients (79% males; median age, 60.0 years), durable complete response (CR) rate was 30%, CR rate was 41%, and objective response rate (CR and partial response) was 75%; 64 patients (80%) achieved hematologic remission. Among complete responders, 27 (85%) achieved MRD negativity by immunohistochemistry. The most frequent adverse events (AEs) were peripheral edema (39%), nausea (35%), fatigue (34%), and headache (33%). Treatment-related serious AEs of hemolytic uremic syndrome (7.5%) and capillary leak syndrome (5%) were reversible and generally manageable with supportive care and treatment discontinuation (6 patients; 7.5%). Moxetumomab pasudotox treatment achieved a high rate of independently assessed durable response and MRD eradication in heavily pretreated patients with HCL, with acceptable tolerability.|
|Molecular Profile||Treatment Approach|
|Gene Name||Source||Synonyms||Protein Domains||Gene Description||Gene Role|
|Therapy Name||Drugs||Efficacy Evidence||Clinical Trials|
|Moxetumomab pasudotox-tdfk||Moxetumomab pasudotox-tdfk||1||0|
|Drug Name||Trade Name||Synonyms||Drug Classes||Drug Description|
|Moxetumomab pasudotox-tdfk||Lumoxiti||CAT-8015||Lumoxiti (moxetumomab pasudotox-tdfk) is an immunotoxin targeting CD22-positive tumor cells (PMID: 30030507). Lumoxiti (moxetumomab pasudotox-tdfk) is FDA approved for use in patients with relapsed or refractory hairy cell leukemia (FDA.gov).|
|Gene||Variant||Impact||Protein Effect||Variant Description||Associated with drug Resistance|
|Molecular Profile||Indication/Tumor Type||Response Type||Therapy Name||Approval Status||Evidence Type||Efficacy Evidence||References|
|Unknown unknown||hairy cell leukemia||not applicable||Moxetumomab pasudotox-tdfk||FDA approved||Actionable||In a Phase III trial that supported FDA approval, Lumoxiti (moxetumomab pasudotox-tdfk) treatment resulted in durable complete response in 30% (24/80), complete response in 41% (33/80), objective response (complete response and partial response) in 75% (60/80), and hematologic remission in 80% (64/80) of patients with relapsed or refractory hairy cell leukemia who had more than 2 prior systemic therapies (PMID: 30030507; NCT01829711).||30030507 detail...|