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Acute Lymphoblastic Leukemia: FDA Expands Besponsa Approval to Include Pediatric Patients

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The US Food and Drug Administration (FDA) has broadened the indication for inotuzumab ozogamicin (marketed as Besponsa by Pfizer) to encompass children aged ≥ 1 year diagnosed with relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia (ALL). Formerly restricted to adults with acute lymphoblastic leukemia, this antibody and cytotoxic drug conjugate now extends its therapeutic reach to pediatric patients.

The pediatric approval of inotuzumab ozogamicin was established upon thorough evaluation within a single-arm study involving 53 children. Among these participants, 12 individuals were initially administered a dose of 1.4 mg/m2 per cycle, while the remaining subjects received an initial dosage of 1.8 mg/m2 per cycle. The study protocol encompassed a median of two treatment cycles, with variations observed in the range spanning one to four cycles per participant.

Safeguarding Children in Clinical Trials: The Critical Role of Premedication Strategies

Before the commencement of acute lymphoblastic leukemia treatment, all enrolled children received premedications as a standard procedure. These premedications included methylprednisolone in combination with antipyretic and antihistamine medications. Such pre-treatment measures are common in clinical trials to mitigate potential adverse reactions and ensure the safety and well-being of study participants throughout the investigation.

By administering these premedications, researchers aimed to minimize the occurrence and severity of adverse events associated with the therapy while maximizing its therapeutic efficacy. This meticulous approach reflects the commitment of healthcare professionals and researchers to uphold the highest standards of patient care and safety in clinical research settings.

Furthermore, the inclusion of premedications underscores the comprehensive nature of the clinical trial process, wherein meticulous attention is paid not only to the efficacy of the investigational acute lymphoblastic leukemia therapy but also to its safety profile and tolerability among study participants. This holistic approach ensures that the data collected from the study are robust, reliable, and reflective of real-world treatment scenarios, thereby contributing to informed clinical decision-making and improving patient outcomes in the long run.

Acute Lymphoblastic Leukemia

Promising Remission Rates in Acute Lymphoblastic Leukemia Trial Despite Adverse Events

Of the cohort, 22 children (42%) achieved complete remission, characterized by < 5% blasts in the bone marrow, absence of leukemia blasts in peripheral blood, full recovery of peripheral blood counts, and resolution of extramedullary disease. The median duration of complete remission stood at 8.2 months. Nearly all children (95.5%) who attained complete remission showed no minimal residual disease (MRD) by flow cytometry, with 86.4% testing MRD negative by real-time quantitative polymerase chain reaction.

Common adverse events, reported in ≥ 20% of participants, encompassed thrombocytopenia, pyrexia, anemia, vomiting, infection, hemorrhage, neutropenia, nausea, leukopenia, febrile neutropenia, increased transaminases, abdominal pain, and headache. In terms of safety, the antibody-drug conjugate carries a black box warning concerning hepatotoxicity, inclusive of hepatic veno-occlusive disease and post-hematopoietic stem cell transplant mortality.

The recommended initial dosage stands at 1.8 mg/m2 per cycle, divided into 0.8 mg/m2 administered on day 1, followed by 0.5 mg/m2 on day 9, and another 0.5 mg/m2 on day 15. For patients experiencing complete remission or complete remission with incomplete hematologic recovery, or to manage toxicities, the initial 3-week cycle may be extended to 4 weeks.


Resource: Med Scape, March 07, 2024

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