Effect of Blinatumomab on KMT2A-rearranged Acute Lymphoblastic Leukemia in Infants
Study design
Single-group study with historical controls from the Interfant-06 trial
Population
- Number of patients studied: 30
- Inclusion criteria: Infants younger than 1 year of age with newly diagnosed KMT2A-rearranged ALL
- Exclusion criteria: Not provided
Interventions
- Experimental group: Interfant-06 chemotherapy with the addition of one postinduction course of blinatumomab
- Control group: Historical controls from the Interfant-06 trial
Primary outcome: 2-year disease free survival %
Adverse Events
| Event Type | Grade 1 | Grade 2 | Grade 3 | Grade 4 |
|---|---|---|---|---|
| Fever | 4 | 0 | 1 | 0 |
| Infection | 0 | 0 | 3 | 1 |
| Hypertension | 0 | 0 | 1 | 0 |
| Vomiting | 0 | 0 | 1 | 0 |
Note: 10 serious adverse events were reported in 9 patients. No fatal adverse events were reported, and no patients had neurologic events. In total, 78 adverse events of any grade occurred. The most frequent grade 3 or higher adverse events were febrile neutropenia, anemia, neutropenia, and elevated γ-glutamyltransferase levels. The most frequent grade 1 or 2 adverse events were hypertension, vomiting, and diarrhea.
Potential Issues and Biases
Short follow-up time, non-randomized design, single-group study design, potential treatment deviations, and limitations in generalizability due to the rarity of the disease.
Conclusion
Blinatumomab added to Interfant-06 chemotherapy appeared to be safe and had a high level of efficacy in infants with newly diagnosed KMT2A-rearranged ALL as compared with historical controls from the Interfant-06 trial.
van der Sluis, I. M., de Lorenzo, P., Kotecha, R. S., Attarbaschi, A., Escherich, G., Nysom, K., Stary, J., Ferster, A., Brethon, B., Locatelli, F., Schrappe, M., Scholte-van Houtem, P. E., Valsecchi, M. G., & Pieters, R. (2023). Blinatumomab added to chemotherapy in infant lymphoblastic leukemia. New England Journal of Medicine, 388(17), 1572-1581.