Background Platelet glycoprotein VI (GPVI) receptor is essential for platelet adhesion and aggreg... more Background Platelet glycoprotein VI (GPVI) receptor is essential for platelet adhesion and aggregation. Eltrombopag is as an effective treatment for chronic immune thrombocytopenia (ITP); yet, its effect on platelet function is not fully characterized. Aim This prospective study investigated the effect of eltrombopag therapy on platelet function through assessment of GPVI receptor expression and soluble GPVI levels among pediatric patients with persistent or chronic ITP. Methods Thirty-six children and adolescents with persistent or chronic ITP were divided equally into two groups either to receive eltrombopag therapy or the standard of care. All patients were followed-up for 12 months with assessment of bleeding score and complete blood count (CBC). Evaluation of GPVI expression using flow cytometry and measurement of its soluble form by ELISA was done at baseline and at 6 months. Results ITP patients on eltrombopag had significantly lower bleeding score after 6 months of therapy w...
Sleep disordered breathing (SDB) is a common underdiagnosed sequela of sickle cell disease (SCD) ... more Sleep disordered breathing (SDB) is a common underdiagnosed sequela of sickle cell disease (SCD) that has been linked to the frequency of vaso-occlusive crises. To determine the frequency of SDB in children with SCD and its association to SCD-related complications, thirty children and adolescents with SCD at their steady state underwent clinical, laboratory, and radiological assessment using transcranial duplex (TCD) and echo assessment of tricuspid regurge velocity (TRV). All participants had an overnight polysomnography after completing the modified STOP-Bang questionnaire. The mean age of the studied cohort was 10.2 years, with male: female ratio 1.7:1. Six children (20%) had high-risk for obstructive sleep apnea (OSA), while nine (30%) were at intermediate risk. Sleep apnea defined as apnea (AHI) > 1 event/hour was found among 18/30 (60%) subjects (14 males and 4 females). Children with AHI > 5 (moderate to severe OSA) had significantly higher TRV (p = 0.007) and left MCA ...
Background Sickle cell retinopathy comprises a constellation of abnormal signs in the retinas of ... more Background Sickle cell retinopathy comprises a constellation of abnormal signs in the retinas of patients with sickle cell disease (SCD). The aim was to detect ophthalmological complications in patients with SCD, particularly macular microvascular alteration, using fundus fluorescein angiography (FFA), and to assess the role of potentially contributory clinicopathological factors including transcranial Doppler, genotypes, hydroxyurea, transfusion therapy, and finally, iron overload state on the development of sickle eye. Patients and methods This cross-sectional study included 30 patients with SCD randomly recruited from the Paediatric Haematology Clinic of Ain Shams University Children’s Hospital, Cairo, Egypt. Complete blood count, transcranial Doppler, and FFA were done. Results Of 30 patients, five had abnormal/conditional transcranial results. The FFA finding was normal in 60 eyes (all 30 SCD patients), and only one patient had abnormal visual acuity. This was a 29-year-old girl who was on simple blood transfusion and hydroxyurea treatment for recurrent cerebral strokes and vaso-occlusive crises, yet her FFA finding was normal. Conclusion The authors could not demonstrate any retinal microvascular alternations using FFA among patients with SCD. The authors related the results to their young age and the fact that all were on hydroxyurea therapy with fair compliance. Further studies using a large sample size are warranted to illustrate the utility of FFA as a tool for early detection of sickle retinopathy.
BACKGROUND Outcome of childhood acute lymphoblastic leukemia (ALL) in low- and middle-income coun... more BACKGROUND Outcome of childhood acute lymphoblastic leukemia (ALL) in low- and middle-income countries is lagging in many aspects including diagnosis, risk stratification, access to treatment and supportive care. OBJECTIVE to report the outcome of childhood ALL at Ain Shams University Children's Hospitals with the use of risk-based protocols before the implementation of minimal residual disease technology and to evaluate the use of double delayed intensification (DDI) in standard risk patients. METHODS Two hundred and twenty patients with ALL diagnosed between January 2005 and December 2014 were included in the study. Patients were treated according to a modified CCG 1991 and 1961 for standard and high risk respectively. Patients were stratified into three risk groups: standard risk (SR), high-risk standard arm (HR-SA), and high-risk augmented arm (HR-AA). RESULTS Among the whole cohort, the 10-year event-free survival (EFS) and overall survival (OS) were 78.1% and 84.3% respectively. Patients with Pre-B immunophenotype (IPT) had significantly better outcome than T-cell IPT (EFS 82.0% versus 58.6%, p < 0.001; OS 86.9% versus 69%, p = 0.003 for Pre-B and T-cell respectively). Among the SR group, patients treated with single delayed intensification (SDI) had comparable EFS and OS rates when compared to patients treated with DDI with EFS 82.4% versus 87.5%, p = 0.825 and OS 88.2% versus 93.5%, p = 0.638 for SDI and DDI groups, respectively. CONCLUSION The use of risk-based protocol with simple laboratory techniques resulted in acceptable survival outcome in resource limited settings. The use of double delayed intensification showed no survival advantage in patients with standard risk.
Background Haemophilic (HF) arthropathy is a major complication in haemophilia. Collagen factors ... more Background Haemophilic (HF) arthropathy is a major complication in haemophilia. Collagen factors are responsible for maintaining the integrity of the vessel wall in the joint. Our goal was to assess the haemophilia joint state and its relation to the clinical, laboratory &radiological data. Methods This cross-sectional study included 50 hemophilia A patients recruited from the Pediatric Hematology / Oncology unit, Children’s Hospital, Ain Shams University,Cairo,Egypt. Assessment of joint status clinicallyand radiologically by X-rays& ultrasound was performed. Results 48% of the studied hemophilia A patients had one, 40 % had two target joints or more. The elbow and knee are the most frequently affected joints (46%,62% respectively).There were no significant differences between inhibitor positive and inhibitor negative patients regarding the number of target joints.However, inhibitor positive patients had significantly lower annual bleeding rate; moreover, none of inhibitor positive ...
Abstract Background: Histiocytoses are unique disorders; their clinical presentations vary from s... more Abstract Background: Histiocytoses are unique disorders; their clinical presentations vary from self-healing lesions to life-threatening disseminated disease. Objectives: We aimed to evaluate the different clinical presentations, frequency of reactivations, and treatment outcome of Langerhans cell histiocytosis among Egyptian children. Methods: we restrospectively analyzed the data of 37 Langerhans cell histiocytosis patients (LCH) registered at Ain Shams University Children’s Hospital for clinicopathological features, treatment modalities and their outcomes. Results: Twenty seven (73%) of the studied patients with LCH had multisystem disease (MS), 24 (88.9%) of them had risk organ involvement (MS RO+) and only 3 without risk organ (MS RO-). Most of the patients received LCH III protocols. Eleven patients (29.7%) had reactivations with median time till reactivation of 17 months (IQR 5-23).Reactivation rates were 40% and 50% in patients with no evidence of active disease (NAD) and those with active disease better (AD better) at week 6 evaluation respectively (p = 0.71).We report 9 deaths (all had MS RO+, two died after reactivation and 7 had progressive disease. The 5 years EFS and OS were 49.4% and 81.2% respectively. Risk stratification did not significantly affect the EFS or OS (p = 0.64 and p = 0.5 respectively). Conclusion: A high reactivation rate was encountered in children with LCH and MS-RO + irrespective of 6 weeks response to induction therapy. A high mortality in patients with progressive disease necessitates a possible earlier aggressive salvage in such group.
The reciprocal of multiecho gradient-echo (ME-GRE) T2* magnetic resonance imaging (MRI) R2*, rise... more The reciprocal of multiecho gradient-echo (ME-GRE) T2* magnetic resonance imaging (MRI) R2*, rises linearly with tissue iron concentration in both heart and liver. Little is known about renal iron deposition in β-thalassemia major (β-TM). To assess renal iron overload by MRI and its relation to total body iron and renal function among 50 pediatric patients with β-TM. Serum ferritin, serum cystatin C, urinary albumin creatinine ratio (UACR), and urinary β2-microglobulin (β2 M) were measured with calculation of β2 M/albumin ratio. Quantification of liver, heart and kidney iron overload was done by MRI. Serum cystatin C, UACR and urinary β2 microglobulin as well as urinary β2m/albumin were significantly higher in β-TM patients than the control group. No significant difference was found as regards renal R2* between Patients with mean serum ferritin above 2500 μg/L and those with lower serum cutoff. Renal R2* was higher in patients with poor compliance to chelation therapy and positively...
We aimed to study the endothelial dysfunction among children and adolescents with transfusion-dep... more We aimed to study the endothelial dysfunction among children and adolescents with transfusion-dependent β-thalassemia using von Willebrand factor antigen (VWF:Ag) and flow cytometric analysis of circulating CD144+ endothelial microparticles (EMPs) and endothelial progenitor cells (CD34+VEGFR2+) and assess their relation to iron overload, erythropoietin and chelation therapy as well as echocardiographic parameters and carotid intima–media thickness. The VWF:Ag, EMPs, and CD34+VEGFR2+ cells were significantly higher among patients with β-thalassemia than controls ( P < .001). The type of chelation and patients’ compliance did not influence the results. No significant correlations were found between the studied vascular markers. Patients with evident heart disease had higher VWF: Ag, EMPs, and CD34+VEGFR2+ cells than those without. Carotid intima–media thickness was increased among patients but not correlated with vascular markers. We suggest that procoagulant EMPs and VWF: Ag are i...
Background Platelet glycoprotein VI (GPVI) receptor is essential for platelet adhesion and aggreg... more Background Platelet glycoprotein VI (GPVI) receptor is essential for platelet adhesion and aggregation. Eltrombopag is as an effective treatment for chronic immune thrombocytopenia (ITP); yet, its effect on platelet function is not fully characterized. Aim This prospective study investigated the effect of eltrombopag therapy on platelet function through assessment of GPVI receptor expression and soluble GPVI levels among pediatric patients with persistent or chronic ITP. Methods Thirty-six children and adolescents with persistent or chronic ITP were divided equally into two groups either to receive eltrombopag therapy or the standard of care. All patients were followed-up for 12 months with assessment of bleeding score and complete blood count (CBC). Evaluation of GPVI expression using flow cytometry and measurement of its soluble form by ELISA was done at baseline and at 6 months. Results ITP patients on eltrombopag had significantly lower bleeding score after 6 months of therapy w...
Sleep disordered breathing (SDB) is a common underdiagnosed sequela of sickle cell disease (SCD) ... more Sleep disordered breathing (SDB) is a common underdiagnosed sequela of sickle cell disease (SCD) that has been linked to the frequency of vaso-occlusive crises. To determine the frequency of SDB in children with SCD and its association to SCD-related complications, thirty children and adolescents with SCD at their steady state underwent clinical, laboratory, and radiological assessment using transcranial duplex (TCD) and echo assessment of tricuspid regurge velocity (TRV). All participants had an overnight polysomnography after completing the modified STOP-Bang questionnaire. The mean age of the studied cohort was 10.2 years, with male: female ratio 1.7:1. Six children (20%) had high-risk for obstructive sleep apnea (OSA), while nine (30%) were at intermediate risk. Sleep apnea defined as apnea (AHI) > 1 event/hour was found among 18/30 (60%) subjects (14 males and 4 females). Children with AHI > 5 (moderate to severe OSA) had significantly higher TRV (p = 0.007) and left MCA ...
Background Sickle cell retinopathy comprises a constellation of abnormal signs in the retinas of ... more Background Sickle cell retinopathy comprises a constellation of abnormal signs in the retinas of patients with sickle cell disease (SCD). The aim was to detect ophthalmological complications in patients with SCD, particularly macular microvascular alteration, using fundus fluorescein angiography (FFA), and to assess the role of potentially contributory clinicopathological factors including transcranial Doppler, genotypes, hydroxyurea, transfusion therapy, and finally, iron overload state on the development of sickle eye. Patients and methods This cross-sectional study included 30 patients with SCD randomly recruited from the Paediatric Haematology Clinic of Ain Shams University Children’s Hospital, Cairo, Egypt. Complete blood count, transcranial Doppler, and FFA were done. Results Of 30 patients, five had abnormal/conditional transcranial results. The FFA finding was normal in 60 eyes (all 30 SCD patients), and only one patient had abnormal visual acuity. This was a 29-year-old girl who was on simple blood transfusion and hydroxyurea treatment for recurrent cerebral strokes and vaso-occlusive crises, yet her FFA finding was normal. Conclusion The authors could not demonstrate any retinal microvascular alternations using FFA among patients with SCD. The authors related the results to their young age and the fact that all were on hydroxyurea therapy with fair compliance. Further studies using a large sample size are warranted to illustrate the utility of FFA as a tool for early detection of sickle retinopathy.
BACKGROUND Outcome of childhood acute lymphoblastic leukemia (ALL) in low- and middle-income coun... more BACKGROUND Outcome of childhood acute lymphoblastic leukemia (ALL) in low- and middle-income countries is lagging in many aspects including diagnosis, risk stratification, access to treatment and supportive care. OBJECTIVE to report the outcome of childhood ALL at Ain Shams University Children's Hospitals with the use of risk-based protocols before the implementation of minimal residual disease technology and to evaluate the use of double delayed intensification (DDI) in standard risk patients. METHODS Two hundred and twenty patients with ALL diagnosed between January 2005 and December 2014 were included in the study. Patients were treated according to a modified CCG 1991 and 1961 for standard and high risk respectively. Patients were stratified into three risk groups: standard risk (SR), high-risk standard arm (HR-SA), and high-risk augmented arm (HR-AA). RESULTS Among the whole cohort, the 10-year event-free survival (EFS) and overall survival (OS) were 78.1% and 84.3% respectively. Patients with Pre-B immunophenotype (IPT) had significantly better outcome than T-cell IPT (EFS 82.0% versus 58.6%, p < 0.001; OS 86.9% versus 69%, p = 0.003 for Pre-B and T-cell respectively). Among the SR group, patients treated with single delayed intensification (SDI) had comparable EFS and OS rates when compared to patients treated with DDI with EFS 82.4% versus 87.5%, p = 0.825 and OS 88.2% versus 93.5%, p = 0.638 for SDI and DDI groups, respectively. CONCLUSION The use of risk-based protocol with simple laboratory techniques resulted in acceptable survival outcome in resource limited settings. The use of double delayed intensification showed no survival advantage in patients with standard risk.
Background Haemophilic (HF) arthropathy is a major complication in haemophilia. Collagen factors ... more Background Haemophilic (HF) arthropathy is a major complication in haemophilia. Collagen factors are responsible for maintaining the integrity of the vessel wall in the joint. Our goal was to assess the haemophilia joint state and its relation to the clinical, laboratory &radiological data. Methods This cross-sectional study included 50 hemophilia A patients recruited from the Pediatric Hematology / Oncology unit, Children’s Hospital, Ain Shams University,Cairo,Egypt. Assessment of joint status clinicallyand radiologically by X-rays& ultrasound was performed. Results 48% of the studied hemophilia A patients had one, 40 % had two target joints or more. The elbow and knee are the most frequently affected joints (46%,62% respectively).There were no significant differences between inhibitor positive and inhibitor negative patients regarding the number of target joints.However, inhibitor positive patients had significantly lower annual bleeding rate; moreover, none of inhibitor positive ...
Abstract Background: Histiocytoses are unique disorders; their clinical presentations vary from s... more Abstract Background: Histiocytoses are unique disorders; their clinical presentations vary from self-healing lesions to life-threatening disseminated disease. Objectives: We aimed to evaluate the different clinical presentations, frequency of reactivations, and treatment outcome of Langerhans cell histiocytosis among Egyptian children. Methods: we restrospectively analyzed the data of 37 Langerhans cell histiocytosis patients (LCH) registered at Ain Shams University Children’s Hospital for clinicopathological features, treatment modalities and their outcomes. Results: Twenty seven (73%) of the studied patients with LCH had multisystem disease (MS), 24 (88.9%) of them had risk organ involvement (MS RO+) and only 3 without risk organ (MS RO-). Most of the patients received LCH III protocols. Eleven patients (29.7%) had reactivations with median time till reactivation of 17 months (IQR 5-23).Reactivation rates were 40% and 50% in patients with no evidence of active disease (NAD) and those with active disease better (AD better) at week 6 evaluation respectively (p = 0.71).We report 9 deaths (all had MS RO+, two died after reactivation and 7 had progressive disease. The 5 years EFS and OS were 49.4% and 81.2% respectively. Risk stratification did not significantly affect the EFS or OS (p = 0.64 and p = 0.5 respectively). Conclusion: A high reactivation rate was encountered in children with LCH and MS-RO + irrespective of 6 weeks response to induction therapy. A high mortality in patients with progressive disease necessitates a possible earlier aggressive salvage in such group.
The reciprocal of multiecho gradient-echo (ME-GRE) T2* magnetic resonance imaging (MRI) R2*, rise... more The reciprocal of multiecho gradient-echo (ME-GRE) T2* magnetic resonance imaging (MRI) R2*, rises linearly with tissue iron concentration in both heart and liver. Little is known about renal iron deposition in β-thalassemia major (β-TM). To assess renal iron overload by MRI and its relation to total body iron and renal function among 50 pediatric patients with β-TM. Serum ferritin, serum cystatin C, urinary albumin creatinine ratio (UACR), and urinary β2-microglobulin (β2 M) were measured with calculation of β2 M/albumin ratio. Quantification of liver, heart and kidney iron overload was done by MRI. Serum cystatin C, UACR and urinary β2 microglobulin as well as urinary β2m/albumin were significantly higher in β-TM patients than the control group. No significant difference was found as regards renal R2* between Patients with mean serum ferritin above 2500 μg/L and those with lower serum cutoff. Renal R2* was higher in patients with poor compliance to chelation therapy and positively...
We aimed to study the endothelial dysfunction among children and adolescents with transfusion-dep... more We aimed to study the endothelial dysfunction among children and adolescents with transfusion-dependent β-thalassemia using von Willebrand factor antigen (VWF:Ag) and flow cytometric analysis of circulating CD144+ endothelial microparticles (EMPs) and endothelial progenitor cells (CD34+VEGFR2+) and assess their relation to iron overload, erythropoietin and chelation therapy as well as echocardiographic parameters and carotid intima–media thickness. The VWF:Ag, EMPs, and CD34+VEGFR2+ cells were significantly higher among patients with β-thalassemia than controls ( P < .001). The type of chelation and patients’ compliance did not influence the results. No significant correlations were found between the studied vascular markers. Patients with evident heart disease had higher VWF: Ag, EMPs, and CD34+VEGFR2+ cells than those without. Carotid intima–media thickness was increased among patients but not correlated with vascular markers. We suggest that procoagulant EMPs and VWF: Ag are i...
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