A Pilot study to assess the safety and efficacy of stem cell therapy in paediatric population with cardiac problems refractory to conventional surgical or medical management
Objectives: Stem cell therapy has been reported to improve cardiac function, reduce pulmonary pressure and improve exercise tolerance in adult patients . The aim of this study is to assess the safety and efficacy of stem cell therapy in patients < 18 years of age with end stage cardiac failure and severe primary or secondary pulmonary hypertension.
Method: 8 patients underwent stem cell therapy. The age ranged from 0.4 to 11 years(Mean age 5.7). Transcoronary implantation of autologous peripheral blood endothelial cells was done in 3 patients with DCM. Transcatheter implantation of autologous bone marrow derived mononuclear cell suspension into RPA and LPA was undertaken in 2 patients with primary pulmonary hypertension and in 1 patient with pulmonary hypertension operated for VSD. Peroperative transmyocardial injection of stem cells was undertaken in 2 infants, the first receiving parent donated peripheral blood endothelial progenitor cells at the time of ALCAPA repair and the second, autologous bone marrow derived mononuclear cells during VSD closure for associated noncompaction of LV.
Results: (As in Table 1)
Conclusion: Stem cell therapy in the pediatric population is safe in the short term period. Intra coronary stem cell implantation for dilated cardiomyopathy results in transient improvement in symptoms and cardiac function. Intrapulmonary stem cell implantation appears to have contributed to significant reduction in pulmonary hypertension in one patient with primary pulmonary hypertension. Intramyocardial implantation of stem cells may have contributed to the improved postoperative cardiac function and recovery in an otherwise high risk substrate of noncompaction of myocardium.
| Diagnosis | Number of patients | Clinical outcome | Follow up details |
|---|---|---|---|
| Dilated Cardiomyopathy | 3 | 2 (Transient improvement of LVEF and reduction of serum BNP at 3 month follow up but failed to sustain the improvement over a longer period.) | No mortality. 1 patient lost to follow-up. |
| Severe primary pulmonary hypertension | 2 | 1 (awaiting repeat cardiac catheterization to assess pulmonary pressure) | 1 mortality. |
| Persisting pulmonary hypertension post VSD surgical closure | 1 | 1 (awaiting repeat cardiac catheterization to assess pulmonary pressure) | No mortality. |
| Anomalous origin of left coronary artery from pulmonary artery | 1 | Significant and rapid resolution of the LV dilatation and dysfunction with transient hypertrophic response of the LV myocardium. | No mortality. |
| Noncompaction of the left ventricle with associated VSD | 1 |