الفهرس 
HEMATOPOIETIC STEM CELL TRANSPLANTATION IN CHILDRENOnly 14 pages are availabe for public view
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Abstract
H
ematopoietic stem cell transplantation (HSCT) is a therapeutic option for numerous hematologic malignant and nonmalignant diseases. Increasing indications for HSCT and improving experience in HSCT and advances in transplantation medicine, has significantly lowered HSCT associated mortality rate, with a consequent increase in the number of long‐term survivors and their long‐term complications. Endocrine complications are among the most prevalent late effects after HSCT. Among those endocrinopathies the metabolic syndrome (MetS) including obesity, hypertension, dyslipidemia, and insulin resistance which place patients at risk of early cardiovascular-related disease. Additionally, growth failure is a frequent complication observed in those survivors.
Metabolic syndrome and growth failure may be related to the patient’s primary illness and its treatment, the nature and intensity of the transplant preparative regimen and graft-versus-host disease (GVHD) and its treatment.
The aim of this study was to assess frequency of MetS and its components in addition to growth failure and their possible risk factors in survivors of HSCT performed during childhood and adolescence that were treated at our center without being exposed to TBI in the conditioning regimen.
This study was conducted on 22 patients who underwent HSCT for different diagnoses between 2011 and 2020 at the pediatric bone marrow transplant (BMT) unit in Ain shams university hospital and survived for more than one year after HSCT: 3 patients were transplanted for malignant diseases, 18 patients for non-malignant diseases and all received chemotherapy only in conditioning with mean age of 12.95 years ranging from 3-21 years, and a mean duration post-transplant of 5.95 years ranging from 1-10 years.
The conditioning regimen drug protocols used in transplanted patients included: Busulfan/Cyclophosphamide (Bu/Cy) in 54.5% of patients, Cyclophosphamide /Antithymocyte globulin (Cy/ATG) in 36.4% of patients, BCNU, etoposide, Ara-c and melphalan (BEAM) in 9.1% of patients.
Two patients received autologous stem cell transplant, while 20 patients received allogeneic stem cell transplant.
All the studied patients were subjected to detailed medical history, complete clinical examination and anthropometric measurements including waist circumference and waist hip ratio, laboratory investigations including:
Complete blood count (CBC), ALT, AST, Serum albumin, BUN, Creatinine, Fasting glucose, Fasting insulin, Homeostatic model assessment of insulin resistance (HOMA IR), HbA1c, Fasting lipid profile, Thyroid function tests and Growth hormone stimulation test for patients with height < -3SD.
The results of the current study were as the following:
• The frequency of metabolic syndrome in our study was 4.5%
• Forty percent had just 2 components of MetS and 18.1% had only one component of MetS.
• Fifty percent of them had hypertriglyceridemia, 45.4% had low HDL-C, 9.1% had elevated blood pressure, 4.5 % had impaired fasting glucose and 4.5% had abdominal obesity.
• None of the patients was obese, 13.6% were overweight and 9% were underweight.
• Non-significant difference between overweight and non-overweight as regards blood pressure Insulin resistance and dysglycemia, Lipid profile and thyroid Function.
• None of the patients was hypertensive, 90.9% were normotensive, 9.1% had elevated blood pressure.
• The frequency of dyslipidemia was 68.2%.
• Non-significant difference between dyslipidemic and non dyslipidemic patients as regards anthropometric measurements, blood pressure GVHD and GVHD treatment, Insulin resistance, dysglycemia and thyroid function.
• Significant difference between dyslipidemic and non dyslipidemic patients as regards serum creatinine and a positive association between creatinine level and dyslipidemia in the multivariate analysis.
• None of our patients was diabetic, 36.4% of our patients had insulin resistance, 4.5% had impaired fasting glucose.
• Non-significant correlation between Fasting glucose and Blood pressure.
• Non-significant correlation between HOMA IR and blood pressure.
• Significant difference between patients with and without insulin resistance as regards acute GVHD treatment.
• Significant positive association between aGVHD and insulin resistance in the multivariate analysis.
• Five from eight patients (5/8) with insulin resistance have received steroids for aGVHD treatment.
• Significant difference between patients with and without insulin resistance as regards Free T3.
• Significant positive association between free T3 and elevated HOMA IR in the multivariate analysis.
• Eighteen percent had short stature (height <-2sd), half of them were <-3sd and had growth hormone deficiency (GHD) related to primary disease.
• Non-significant difference between patients with and without short stature as regards GVHD and GVHD treatment, Insulin resistance, dysglycemia, Lipid profile and Thyroid Function.
• No variable was associated with short stature in the multivariate regression analysis.
• None of patients with short stature had obesity, abdominal obesity, dysglycemia, insulin resistance or abnormal thyroid function.
• Seventy five percent of short patients had high LDL, 50 % had low HDL_C, 50% had high total cholesterol and 25% had high TG.
• Thyroid disorders found in 22.7% of patients, 13.6% having subclinical hypothyroidism and 9.1% having hyperthyroidism.
• In conclusion, we confirmed a high prevalence of MetS components, specifically dyslipidemia (68.2%) and insulin resistance (36.4%) among HSCT survivors despite non exposure to irradiation.
• Short stature was found in 18.2% of patients and 9.1% were found to have growth hormone deficiency with the most important contributor to growth failure being the primary disease.
• Screening and management of MetS and Growth retardation should be integrated within models of routine long-term follow-up care after HSCT.