Mesenchymal stem cell therapy as adjunctive treatment in pediatric patients with severe acquired brain injury: a single-center retrospective case series - Summary - MDSpire

Mesenchymal stem cell therapy as adjunctive treatment in pediatric patients with severe acquired brain injury: a single-center retrospective case series

  • By

  • Hacer Uçmak

  • Merve Havan

  • Anar Gurbanov

  • Burak Balaban

  • Fevzi Kahveci

  • Hasan Özen

  • Edin Botan

  • Emrah Gün

  • Ayşen Durak Aslan

  • Eda Eyduran

  • Ömer Bektaş

  • Ayşe Eser Elçin

  • Tanıl Kendirli

  • July 14, 2026

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Objective:

To describe the clinical course and short-term neurological outcomes of pediatric patients who received MSC therapy as an adjunct to conventional intensive care.

Approach:
  • Study Design: Single-center retrospective case series including pediatric patients who received MSC therapy in the PICU from January 2019 to August 2023.
  • Patient Characteristics: Included 6 patients (5 males, 1 female; median age 65.5 months) with severe acquired brain injury.
  • MSC Administration: Allogeneic MSCs were administered at a dose of 1–2 × 10^6 cells/kg per session over 3–5 sessions, intravenously and/or intrathecally.
  • Outcome Assessment: Neurological outcomes were assessed using the Glasgow Coma Scale (GCS) and Pediatric Cerebral Performance Category (PCPC) at baseline, 1 month, and 6 months after MSC therapy.
Key Findings:
  • Median GCS improved from 4 (range 3–5) before MSC therapy to 11 (range 8–15) after treatment.
  • PCPC scores decreased from a median of 5 (range 5–5) at baseline to 4 (range 4–4) at 1 month and 3 (range 3–3) at 6 months.
  • No procedure-related adverse events were observed during MSC administration or follow-up.
  • All six patients survived.
Interpretation:

Neurological improvement was observed during follow-up; however, due to small sample size, lack of control group, and heterogeneous protocols, improvement cannot be solely attributed to MSC therapy.

Limitations:
  • Small sample size.
  • Lack of a control group.
  • Heterogeneous etiologies and treatment protocols.
  • Concomitant conventional care and rehabilitation.
Conclusion:

Adjunctive MSC therapy was feasible and well tolerated in pediatric patients with severe acquired brain injury, with observed neurological improvement.

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