Romiplostim stimulates thrombopoietin receptor to increase platelet production of megakaryocytes in idiopathic thrombocytopenic purpura (ITP). This study aimed to evaluate the safety and efficacy of romiplostim in children with chronic ITP. Eighteen patients with chronic ITP, either none responsive or failed to maintain response on two or more therapeutic modalities, were enrolled. Patients were randomized (2:1) to receive romiplostim or placebo for 12 weeks, initiated at 1 μg/kg/week, escalated to 5 μg/kg/week, and then tapered. Median patients' age was 8.5 years, and the median baseline platelet count (PC) was 10.5 × 10(9)/L. The median weekly dose of romiplostim was 2 μg/kg. Fifty percent of patients in both romiplostim and placebo arms had at least one adverse event (AE); none was serious. Ten patients on romiplostim (83.3%) maintained the efficacy endpoint (PC > 50,000). Romiplostim was well-tolerated and efficient in treating the children with chronic refractory ITP with no unexpected AEs.

Mutations in glucokinase (GCK) cause a spectrum of glycemic disorders. Heterozygous loss-of-function mutations cause mild fasting hyperglycemia irrespective of mutation severity due to compensation from the unaffected allele. Conversely, homozygous loss-of-function mutations cause permanent neonatal diabetes requiring lifelong insulin treatment. This study aimed to determine the relationship between in vitro mutation severity and clinical phenotype in a large international case series of patients with homozygous GCK mutations. Clinical characteristics for 30 patients with diabetes due to homozygous GCK mutations (19 unique mutations, including 16 missense) were compiled and assigned a clinical severity grade (CSG) based on birth weight and age at diagnosis. The majority (28 of 30) of subjects were diagnosed before 9 months, with the remaining two at 9 and 15 years. These are the first two cases of a homozygous GCK mutation diagnosed outside infancy. Recombinant mutant GCK proteins were analyzed for kinetic and thermostability characteristics and assigned a relative activity index (RAI) or relative stability index (RSI) value. Six of 16 missense mutations exhibited severe kinetic defects (RAI ≤ 0.01). There was no correlation between CSG and RAI (r2 = 0.05, P = 0.39), indicating that kinetics alone did not explain the phenotype. Eighty percent of the remaining mutations showed reduced thermostability, the exceptions being the two later-onset mutations which exhibited increased thermostability. Comparison of CSG with RSI detected a highly significant correlation (r2 = 0.74, P = 0.002). We report the largest case series of homozygous GCK mutations to date and demonstrate that they can cause childhood-onset diabetes, with protein instability being the major determinant of mutation severity.

Background The COVID-19 pandemic and the consequences of lockdown significantly impacted glycemic control. Aim To evaluate the impact of the pandemic and lockdown on glycemic control among Egyptian children and adolescents with type 1 diabetes. Methods Cross-sectional study conducted through an online questionnaire. The participants were patients with type 1 diabetes and/or their caregivers Results A total of 115 valid responses to the questionnaire were received. During the lockdown, almost 64% of patients showed worsening of their HbA1C with significant increment of HbA1c after the lockdown (p < 0.001). Synchronous simple telemedicine service was initiated through phone calls and social media applications, and 97% of the patients and their families were successfully able to continue follow-up. Almost 76% of the patients/caregivers showed moderate stress which was significantly correlated with HbA1C (p < 0.05). Fear of hospital admission and fear from shortage of medical supplies were the main COVID-19-related worries. Conclusion The lockdown negatively impacted glycemic control and initiated a set of COVID-19 worries and stress among patients and their caregivers in Egypt. Telemedicine service, even simple tools, is effective and important for the continuity of care among patients. The limited availability and the fear of shortage of medical supply forced patients to ration glucose monitoring.

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Background: Coronavirus disease-2019 (COVID-19) could be associated with morbidity and mortality in immunocompromised children. Objective: To measure the frequency of SARS-COV-2 infection among hospitalized children with cancer and detect the associated clinical manifestations and outcomes. Methodology: A prospective non-interventional study including all hospitalized children with cancer conducted between mid-April and mid-June 2020 in Ain Shams University hospital, Egypt. Clinical, laboratory and radiological data were collected. SARS-CoV2 infection was diagnosed by RT-PCR tests in nasopharyngeal swabs. Results: Fifteen of 61 hospitalized children with cancer were diagnosed with SARS-COV-2. Their mean age was 8.3±3.5 years. Initially, 10(66.7%) were asymptomatic and 5(33.3%) were symptomatic with fever and/or cough. Baseline laboratory tests other than SARS-COV-2 RT-PCR were not diagnostic; the mean absolute lymphocyte count was 8.7±2.4 x109/L, C-reactive protein was mildly elevated in most of patients. Imaging was performed in 10(66.7%) patients with significant radiological findings detected in 4(40%) patients. Treatment was mainly supportive with antibiotics as per the febrile neutropenia protocol and local Children Hospital guidance for management of COVID-19 in children. Conclusion: Pediatric cancer patients with COVID-19 were mainly asymptomatic or with mild symptoms. A high index of suspicion and regular screening with nasopharyngeal swab in asymptomatic hospitalized cancer patients is recommended. Introduction: Coronavirus disease 2019 (COVID-19) is a viral respiratory illness caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the first case was detected in December 2019. (1) The COVID-19 pandemic has rapidly escalated into a global crisis. (2) The first case registered in Egypt was in February 2020 and since that date, the numbers have been increasing and by mid-June 2020, 47,856 cases of SARS-CoV-2 and 1766 deaths have been reported by the Ministry of Health (3). Children are usually develops mild disease (4), a Chinese analysis of 2135 confirmed or suspected cases of COVID-19 among children revealed that 55.4% developed mild symptoms or were asymptomatic and only 5.4% had severe illness compared with 18.5% of adult cases. (5) Viral infections in general are associated with increased morbidity and mortality in immunocompromised children. (6) Data on the clinical features and outcomes of immunocompromised children with cancer who are infected with SARS-CoV-2 are scarce. Knowledge from previous influenza A H1N1 pandemics suggests that those vulnerable immunosuppressed children are likely to be infected and to develop manifestation as an increased number of cases are described with time. (7,8) Although isolation as an inpatient is common practice for children who are receiving intensive chemotherapy or stem cell transplant, most children with cancer are treated in the outpatient setting, and hospital visits