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Published on: July 10, 2019
Study of peripheral stem cells mobilization as a treatment line of pediatric dilated cardiomyopathy
Mohamed El-Shanshory1, Waled El-Shehaby1, Nahed Hables1
11 Department of Pediatrics, 2 Department of Clinical Pathology, 3 Department of Cardiology, Faculty of Medicine, Tanta University, Tanta, Egypt.
Insights
Granulocyte-colony stimulating factor (G-CSF) therapy improved heart function in children with idiopathic dilated cardiomyopathy (IDCM). This treatment mobilized stem cells and enhanced systolic function, showing promise for pediatric heart conditions.
Area of Science:
- Pediatric Cardiology
- Hematology
- Regenerative Medicine
Background:
- Idiopathic dilated cardiomyopathy (IDCM) in children presents a significant clinical challenge.
- Mobilizing hematopoietic stem cells is a potential therapeutic strategy for pediatric IDCM.
- Granulocyte-colony stimulating factor (G-CSF) is investigated for its efficacy in this context.
Purpose of the Study:
- To evaluate the therapeutic efficacy of G-CSF in pediatric patients diagnosed with IDCM.
- To assess the impact of G-CSF on hematopoietic stem cell mobilization and cardiac function.
Main Methods:
- A randomized clinical trial involving 40 pediatric IDCM patients.
- Assessment of clinical status, cardiac biomarkers (LDH, CPK, CK-MB), and peripheral blood CD34(+) cells.
- Echocardiography and G-CSF subcutaneous injections over 5 consecutive days.
Main Results:
- Patients showed clinical improvement, including regression in heart failure classification.
- G-CSF administration led to increased CD34(+) cell mobilization and elevated white blood cell counts.
- Significant improvements were observed in echocardiographic parameters of systolic function (ejection fraction, fractional shortening, Sm).
Conclusions:
- G-CSF administration appears beneficial for enhancing systolic heart function in pediatric IDCM.
- Further research with larger patient cohorts is warranted to confirm these findings.
- G-CSF shows potential as a therapeutic agent in pediatric cardiovascular diseases.
Background:
Mobilizing hematopoietic stem cells may be a promising intervention for the treatment of idiopathic dilated cardiomyopathy (IDCM) in infant and children. So the aim of the work is to evaluate the efficacy of granulocyte-colony stimulating factor (G-CSF) as a therapeutic modality in pediatric IDCM.
Methods:
A randomized clinical trial was conducted on 40 pediatric patients with IDCM. They were subjected to history taking, clinical examination, serum lactate dehydrogenase (LDH), total creatinine phosphokinase (CPK), creatinine phosphokinase isoenzyme B (CK-MB) isoenzyme, and peripheral blood CD34(+) cell assessment before and at day 7 after subcutaneous G-CSF injection for 5 consecutive days. Echocardiography was done before and 1, 3 and 6 months after therapy.
Results:
Clinical improvement in the form of regression of patients Modified Ross heart failure (MRHC) classification classes. Increased percentage of CD34(+) mobilized cells from the bone marrow, and significant increase in blood counts especially white blood cells 7 days after G-CSF injection. Significant improvement was found in echocardiographic data evaluating systolic function of the heart [Ejection fraction, Fractional shortening and systolic velocity at mitral annulus (Sm)].
Conclusions:
Administration of G-CSF may be beneficial in improving systolic functions of the heart in pediatric IDCM and more studies with a large number of patients are needed.
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