First Results of using Stem Cell Transplantation for Pediatric Patients in Case of Dilated Cardiomyopathy (original) (raw)

A Study of Intracoronary Injection of Hematopoietic Stem Cells in Pediatric Dilated Cardiomyopathy: Is It an Applicable Solution for Critically Ill Patients?

Iranian Journal of Pediatrics

Background: Benefits of stem cell therapy on remodelling and cardiac function have been described in adults with dilated cardiomyopathy and acute myocardial infarction. Objectives: We investigated the effect of this treatment modality amongst children with severe dilated cardiomyopathy. Methods: Intracoronary injection of autologous bone marrow mononuclear stem cells was performed in our centers for 8 severely ill children during 2015-2016. The mean age of the patients was 10.1 years (5 girls, 3 boys). They were followed by longitudinal speckle tracking echocardiography (STE) and conventional echocardiography for 6 months. Results: Heart functional class improved in 62% of patients. M-mode echocardiography showed significant improvement in ejection fraction (mean 24.8 ± 8.3 vs. 37.4 ± 10.5) and in STE, the mean global longitudinal strain improved (GLS:-2.8 ± 1.9 vs.-5.2 ± 3.9). None of the patients had serious complications. Conclusions: Intracoronary injection of autologous mononuclear stem cells might improve the ventricular function and cardiac remodelling in pediatric patients with dilated cardiomyopathy and could be considered in critically ill patients.

Clinical Study Therapeutic Role of Mobilized Bone Marrow Cells in Children with Nonischemic Dilated Cardiomyopathy

2012

Copyright © 2012 Nevin M. Habeeb et al. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Dilated cardiomyopathy is an important cause of congestive cardiac failure in infants and children. Mobilizing hematopoietic progenitor cells is a promising intervention to this deadly disease. Aim. Evaluate granulocyte colony stimulating factor (GCSF) as therapeutic modality in children with idiopathic dilated cardiomyopathy (IDCM). Subjects and Methods. This case-control prospective study was conducted on 20 children with IDCM following up at Cardiology Clinic Children’s Hospital, Ain Shams University (group 1) who were compared to another 10 age-, sex-, duration-of-illness-, and systolic-function-matched children with IDCM as control (group 2). They were subjected to history taking, clinical examination, echocardiography, and per...

Therapeutic Role of Mobilized Bone Marrow Cells in Children with Nonischemic Dilated Cardiomyopathy

ISRN Pediatrics, 2012

Dilated cardiomyopathy is an important cause of congestive cardiac failure in infants and children. Mobilizing hematopoietic progenitor cells is a promising intervention to this deadly disease. Aim. Evaluate granulocyte colony stimulating factor (GCSF) as therapeutic modality in children with idiopathic dilated cardiomyopathy (IDCM). Subjects and Methods. This case-control prospective study was conducted on 20 children with IDCM following up at Cardiology Clinic Children's Hospital, Ain Shams University (group 1) who were compared to another 10 age-, sex-, duration-of-illness-, and systolic-function-matched children with IDCM as control (group 2). They were subjected to history taking, clinical examination, echocardiography, and peripheral blood CD34+ cell assessment before and one week after GCSF intake for 5 consecutive days (by group 1 but not group 2). Results. A significant improvement in echocardiographic data and CD34+-T-cell increase was found in group 1 one week after GCSF intake and for the next 6 months CD34+ T cells percentage of change showed no significant correlation with the that of the left ventricular dimensions and systolic function. Conclusion. Administration of GCSF to children with IDCM resulted in clinical and echocardiographic improvement not correlated to mobilized CD34+ T cells, implying involvement of additional mechanisms over simple stem cell mobilization.

Management of Idiopathic Dilated Cardiomyopathy with Intramyocardial Stem Cell Transplantation in Children: A Retrospective Study of 7 Patients

Science Journal of Clinical Medicine, 2013

This retrospective report presents findings on 7 patients with idiopathic dilated cardiomyopathy who underwent transplantation of autologous bone marrow derived mononuclear cells (BMSC) after failed routine conservative treatment, and were on a waiting list for cardiac transplantation. In two out of seven patients, we performed intramyocardial delivery of BMSC twice, 22 and 24 months respectively after the first transplant. All patients received anti-congestive therapy by diuretics, ACE inhibitors, carvedilol, digitalis and aspirin. We discuss potential disadvantages of intracoronary injections of autologous bone marrow stem cells. We believe that our BMSC transplantation method as opposed to intravascular input is the method of choice, particularly in children with idiopathic dilated cardiomyopathy because it is safe, gentle and is not associated with risks for coronary circulation.

Transcutaneous intramyocardial injection technique for implantation ofbone marrow derived stem cells in children with idiopathic dilated cardiomyopathy

2014

An increasing understanding of the nature and processes of idiopathic dilated cardiomyopathy in children, as well as the limited treatment options have led several researchers to use stem cell transplantation in management of these patients. Study results suggest that homing of transplanted cells varies significantly using different techniques for their delivery to the target area. We will describe in detail the technique for transdermal intramyocardial implantation of bone marrow derived progenitor cells. This innovative technique if combined with ultrasound monitoring offers the possibility for delivery of stem cells right into the target area in a safe and effective way.

Safeguards and Pitfalls in Technique Used for Stem Cell Delivery in Children Suffering from Idiopathic Dilated Cardiomyopathy

Journal of US-China Medical Science, 2013

We report 7 pediatric patients with idiopathic dilated cardiomyopathy who underwent autologous bone marrow derived mononuclear cells intramyocardial delivery as a final option after unsuccessful routine conservative treatment and have real perspective for cardiac transplantation in childhood in our country at new time. For two patients intramyocardial delivery was performed repeatedly 22 and 24 months respectively after first repair. All patients received massive anti-congestive therapy by diuretics, ACE inhibitors, carvedilol, digitalis and aspirin.

Resultados de un programa nacional de trasplante cardiaco pediátrico: fortalezas y debilidades

Revista chilena de pediatría, 2017

Introduction: Pediatric heart transplantation is an effective therapy to treat advanced heart failure in children. Objectives: To analyze the immediate and mid-term results of pediatric patients listed for heart transplantation. Material and Methods: Registration of patients admitted to our transplant protocol between October 2001 and July 2016 were reviewed, analyzing demographic data, diagnosis, status at the time of listing, waiting time until transplantation, donor data, use of ventricular assist device, hemodynamic data, complications and global mortality. Results: Thirthy patients where included with a mean age of 9.4 years (1 month to 15 years). The most frequent diagnosis was dilated cardiomyopathy in 24 patients (80%). The status was I (urgency) in 19 cases and II in 11 cases. Ten patients died on the waiting list (33,3%) at an average of 52 days (13-139 days). Fourteen were transplanted (46.7%), with a waiting time of 199.6 days (4-586 days). Nine patients required mechanical support (30%). All patients received triple association of immunosuppression. One patient died 16 days post transplant due to primary graft failure (7.1%). The average follow-up was 43 months (0.5-159 months). Two patients died later on (82 and 55 months), both due to secondary rejection because of voluntary cessation of immunosuppressive therapy. Survival at 1 and 5 years was 93% and 74%, respectively. Conclusions: Our program has successfully transplanted 50% of patients enrolled, with good medium-term survival. A significant proportion of patients were listed urgently and 34.5% died on the waiting list.

[Cell therapy in dilated cardiomyopathy]

Arquivos brasileiros de cardiologia, 2006

A forty-one-year-old male with systolic heart failure, FC-III NYHA, clinical stage C due to dilated cardiomyopathy was submitted to an autologous transplant of the mononuclear fraction of bone marrow via coronary artery system through heart catheterism. Two months after the procedure, there was a decrease in plasma BNP and cardiac area reduction at the thorax X-ray and nuclear magnetic resonance. The echocardiogram showed decrease of the secondary regurgitation and mitral ring dilatation. There was a better performance at the ergospirometry, with increase of the maximum oxygen consumption and consequent reduction in drug therapy. The absence of adverse events characterized by clinical/hemodynamic instability, enzymatic alteration or electrocardiogram demonstrate the safety and feasibility of this procedure carried out and described with pioneering spirit in dilated cardiomyopathy.

Transplantation of bone marrow stem cells during cardiac surgery

Asian Cardiovascular and Thoracic Annals, 2014

Background: This systematic review with meta-analysis sought to determine the efficacy and safety of intramyocardial transplantation of bone marrow stem cells during coronary artery bypass graft surgery on postoperative cardiac functional parameters such as left ventricular ejection fraction and left ventricular end-diastolic volume. Methods: Medline/PubMed, Embase, Elsevier, Sciences online database, and Google Scholar literature search were searched. The effect sizes measured were risk ratio for categorical variables and weighted mean difference with 95% confidence interval for calculating differences between mean values of baseline and follow-up cardiac functional parameters. A value of p < 0.1 for Q test, or I 2 > 50%, indicated significant heterogeneity among studies. The literature search retrieved 2900 studies from screened databases, of which 2866 (98.6%) were excluded and 34 (619 patients) were included for scoping review. The final analysis included 9 studies (335 patients). Results: Pooled effects estimates of left ventricular ejection fraction and left ventricular end-diastolic volume showed that bone marrow stem cell transplantation had a weighted mean difference of 4.06 (95% confidence interval: 0.41-7.72; p ¼ 0.02) and 7.06 (95% confidence interval: À8.58-22.7; p ¼ 0.3), respectively. Conclusions: Intramyocardial transplantation of bone marrow stem cells improves cardiac functional parameters, significantly increasing left ventricular ejection fraction with a nonsignificant reduction in left ventricular end-diastolic volume. Also, this therapeutic method has no life-threatening complications and was therefore found to be an effective and safe method.