Journal: Journal of clinical oncology : official journal of the American Society of Clinical Oncology
Phase II/retrospective-comparative pediatric ALL study (infants with KMT2A‑rearranged ALL)
Population & Design
- – 30 infants with KMT2A‑rearranged acute lymphoblastic leukemia received standard Interfant‑06 chemotherapy plus one post‑induction course of blinatumomab.
- – Outcomes were compared with a historical, similarly treated Interfant‑06 cohort that did not receive blinatumomab.
- – Median follow‑up: 4.2 years (range 3.2–6.0).
Efficacy
- – 4‑year disease‑free survival: 83.3% with blinatumomab vs 44.0% in historical controls.
- – 4‑year overall survival: 93.3% with blinatumomab vs 60.2% in historical controls.
- – These data indicate a sustained long‑term benefit when blinatumomab is added post‑induction.
Safety & Supportive Care
- – No infection‑related deaths occurred post‑induction in the blinatumomab group (historical Interfant‑06 had ~4% infection‑related mortality).
- – Intravenous immunoglobulin was given in 19/30 (63%); G‑CSF in 5/30 (17%).
- – No new long‑term safety signal is described in the abstract; infectious toxicity appears manageable.
Pharmacokinetics
- – Mean steady‑state concentration (Css): 706 ± 194 pg/mL/day.
- – Median clearance: 0.89 L/h/m² (range 0.57–2.66).
- – Pharmacokinetic profile is comparable to that observed in older pediatric populations.
Clinical Takeaway
- – Incorporating a single post‑induction course of blinatumomab into an Interfant‑based regimen for infants with KMT2A‑rearranged ALL produced a marked and durable improvement in DFS and OS with acceptable toxicity and predictable PK.
- – These results support blinatumomab as a promising component of frontline therapy in this very high‑risk subgroup, pending confirmation in prospective ongoing trials.