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1.
Front Pediatr ; 8: 564, 2020.
Artigo em Inglês | MEDLINE | ID: mdl-33014944

RESUMO

Aim: Anoxic brain injury (ABI) due to non-fatal drowning may cause persistent vegetative state (VS) that is currently incurable. The aim of this paper is to present the safety and feasibility of autologous bone marrow-derived mononuclear cell (BMMNC) transplantation in five drowning children surviving in persistent VS. Methods: We used BMMNC as a novel candidate therapeutic tool in a pilot phase-I study for five patients affected by neurological sequelae after near-death drowning. Autologous BMMNCs were freshly isolated using Ficoll gradient centrifugation then infused intrathecally to five patients. The number of transplantation varied from two to four times depending on the motor function improvement of patient after transplantation. Clinical therapeutic effects were evaluated using gross motor function measure and muscle spasticity rating scales, cognitive assessments, and brain MRI before and after cell administrations. Results: Six months after BMMNC transplantation, no serious complications or adverse events were reported. All five patients displayed improvement across the major parameters of gross motor function, cognition, and muscle spasticity. Three patients displayed improved communication including the expression of words. In particular, one patient remarkably reduced cerebral atrophy, with nearly normal cerebral parenchyma after BMMNC transplantation. Conclusions: Autologous BMMNC transplantation for the treatment of children in persistent VS after drowning is safe, feasible, and can potentially improve motor function and cognition and reduce muscle spasticity. These results pave the way for a future phase II clinical trial to evaluate the efficacy of the therapy.

2.
Am J Case Rep ; 19: 1010-1018, 2018 Aug 25.
Artigo em Inglês | MEDLINE | ID: mdl-30143601

RESUMO

BACKGROUND Bowel dysfunction is observed in 42.2-71.2% of patients with spina bifida. Traditional treatments yield limited results. The objective of this paper is to report on improvement in bowel function in 2 children with spina bifida following bone marrow-derived mononuclear cells transplantation. CASE REPORT Two patients - 14 years old and 11 years old - with bowel dysfunction after myelomeningocele repair underwent 2 BMMNC transplantations without complications. Those patients had normal defecation, assessed through follow-ups of 21 months and 16 months, respectively. CONCLUSIONS BMMNC transplantation can improve bowel function, as demonstrated in 2 patients with spina bifida.


Assuntos
Transplante de Medula Óssea , Constipação Intestinal/terapia , Disrafismo Espinal/terapia , Adolescente , Criança , Constipação Intestinal/etiologia , Feminino , Humanos , Masculino , Disrafismo Espinal/complicações
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