Children's Medical Research Articles

Children's perspective of hope in living with leukemia: A qualitative study using drawing

PurposeThis study aimed to explore children's perspective of hope in living with leukemia through the utilization of drawing-based interview technique. Design and methodsThis qualitative study was conducted at the Children's Medical Center in Tehran with the participation of children aged 6 to 12 diagnosed with leukemia. The children were asked to prepare a drawing about hope then interviews were conducted face-to-face with each child after completing the drawing. Data analysis was conducted using the qualitative content analysis. ResultsIn the collection of a total of 20 drawings from school-age children with leukemia, each drawings has different metaphors symbolizing hope. Five themes emerged including positive thinking, embracing the sanctuary of maternal affection and collective caring, the ethereal glow of joy, the melody of resilient soul, and navigating the way back to home. ConclusionHope from the child's perspective can be achieved in simple, possible and accessible ways, making it easier for the family, community and healthcare system to inspire hope in their lives with Leukemia. Practice implicationHealth providers in pediatric oncology can integrate hope-based care interventions related to the factors influencing children's perspectives.

Mothers' Experiences of Children Undergoing Congenital Heart Disease Surgery: A Qualitative Study

Background: Surgery for children with congenital heart disease (CHD) elevates maternal physical and mental stress, potentially impacting the quality of care provided to the child. Objectives: This study aimed to elucidate mothers' experiences surrounding their child's CHD surgery. Methods: In this qualitative content analysis study, we included 20 mothers of children admitted to Tehran Children's Medical Center Hospital for CHD surgery using purposive sampling. Unstructured interviews and field notes were utilized perioperatively for data collection. Analysis was conducted concurrently with data collection employing the Graneheim and Lundman content analysis approach. Rigor was ensured using Lincoln and Guba’s criteria. Results: The findings were categorized into four themes: (1) "The Bitter and Difficult Motherhood Experience" stemming from the shock of the child's abnormality and subsequent challenges in caregiving; (2) mothers engaged in "In Search of Reassurance," striving for accurate diagnosis and optimal treatment quality through information seeking and accessing superior medical services; (3) "Tides in Supportive Networks" reflected varying degrees of support from personnel, peers, and surrounding individuals, ranging from conscientiousness to stressfulness; (4) Mothers attained a state of "Relative Calm After the Storm" by navigating through the surgery ordeal with spirituality and hope despite enduring anxieties. Conclusions: The journey through a child's CHD surgery proved harrowing for mothers due to the initial shock and ongoing caregiving challenges. Care provision should prioritize maternal readiness and empowerment, emphasize professional conduct, reassurance, spiritual care, and steer social support networks towards positive contributions.

Tardive Dyskinesia with Antipsychotic Medication in Children and Adolescents: A Systematic Literature Review.

Tardive dyskinesia (TD) is a persisting, and potentially irreversible, movement disorder associated with treatment with dopamine receptor antagonists. Few data are available on the risk of TD in children and adolescents treated with antipsychotic medication. To review the literature on incidence, risk factors, and treatment options for antipsychotic-associated TD in children and adolescents (aged < 18 years). Relevant articles were identified through a systematic search of Embase and Medline performed in January 2024. Methodological quality was assessed using the Newcastle-Ottawa Scale and Joanna Briggs Institute Critical Appraisal tools. Thirteen studies were identified. The reported TD point prevalence was 5-20%, with higher rates in studies involving typical antipsychotics. Lower estimates (around 1%) emerged from analyses of clinical database data suggesting underdiagnosis in clinical practice. Risk factors included treatment with typical antipsychotics, higher doses, longer duration of exposure, older age, female gender, higher baseline Abnormal Involuntary Movements Scale (AIMS) scores, intellectual impairment, and perinatal complications. Although relatively few cases have been reported in children and adolescents, TD remains a risk in this population. Individuals receiving antipsychotics should be monitored carefully for the emergence of abnormal movements. Other than dose reduction, discontinuation, or switch to a lower-risk antipsychotic, few interventions have demonstrated efficacy. The strongest evidence for pharmacological treatment is for VMAT-2 inhibitors (valbenazine and deutetrabenazine), but these drugs are not licensed for use in children. To reduce risk, antipsychotics should be prescribed only if necessary, at the minimum effective dose and for the minimum necessary duration.

The Impact of Stimulant Medication on the Relation Between Working Memory and Activity Level in ADHD.

There is a positive association between heightened activity levels and improved working memory performance (WM) in individuals with Attention-Deficit/Hyperactivity Disorder (ADHD). Recent research suggests that stimulant medications may have a simultaneous positive impact on WM and motor skills. Yet, it is unclear the specific connection between movement, WM, and stimulant use. We examined how visuospatial (VS) and phonological (PH) WM performance varied with children's stimulant medication usage and naturally occurring activity level. In a repeated measures design, children with ADHD (n = 43; 7-12years old) completed WM tasks while wearing actigraphy watches to monitor activity level on and off stimulant medication. Significant large sized main effects were observed for medication condition on PH (p < .05, ηp2 = .14) and VS (p < .001, ηp2 = .30) WM. Activity level also had significant medium sized main effects on PH (p < .01, ηp2 = .09) and VS (p < .005, ηp2 = .10) WM. There was a significant medium sized interaction for VS WM (p < .005, ηp2 = .11), indicating that the effect of medication on performance was greatest in the highest activity level category. The findings suggest that a combination of stimulant medication and an "optimal" level of movement may be most effective for improving VS WM.

Paediatric Drug Development in China: Current Status and Future Prospects.

For more than two decades, regulatory agencies throughout the world released guidelines, rules and laws to stimulate and assist in paediatric drug development. In 2014, the National Health and Family Planning Commission (now known as the National Health Commission, NHC) and five other departments in China jointly issued 'Several Opinions on Safeguarding Medication for Children', after which several policies and regulations were issued to implement the priority review and approval of paediatric medicinal products and support the development of new drugs, including new dosage forms and strengths, for children. A total of 172 special medicinal products for children were approved from 2018 to 2022. Since 2016, the NHC, together with relevant administrative departments, has formulated and issued four paediatric drug lists containing 129 medicinal products to encourage research and development. At present, approximately 25 of these drugs (at exactly the same dosage forms and strengths as on the lists) have been approved for marketing, including antitumour drugs and immunomodulators, nervous system drugs, drugs for mental disorders and drugs for rare diseases. In this review, we analysed the regulations issued for promoting paediatric drug development in China, including the priority review and approval system, technical guidelines, data protection and financial support policies and general profiles of paediatric drug approval, clinical trials and the addition of information for children in the labels of marketed medicinal products. Finally, we discussed the challenges and possible strategies in the research and development of paediatric drugs in China.

Therapeutic plasma exchange as treatment modality in pediatric patients with neurologic and nonneurologic diseases at the Philippine Children's Medical Center: A 12‐year single center experience

Therapeutic plasma exchange as treatment modality in pediatric patients with neurologic and nonneurologic diseases at the Philippine Children's Medical Center: A 12‐year single center experience

Child and Adolescent Mental Health Service (CAMHS) Black Country Healthcare Foundation Trust (BCHFT) Trust-Wide Audit on Adherence to NICE Guidelines in Prescribing Medications for Children With Autism Spectrum Disorder (ASD)

Child and Adolescent Mental Health Service (CAMHS) Black Country Healthcare Foundation Trust (BCHFT) Trust-Wide Audit on Adherence to NICE Guidelines in Prescribing Medications for Children With Autism Spectrum Disorder (ASD)

Isolation, Identification and Antibiotic Resistance of Anaerobic Bacteria in Sputum of Cystic Fibrosis Patients from Children's Medical Center of Tehran, Iran

Isolation, Identification and Antibiotic Resistance of Anaerobic Bacteria in Sputum of Cystic Fibrosis Patients from Children's Medical Center of Tehran, Iran

Reducing postoperative hypothermia in infants: Quality improvement in China.

Unintended postoperative hypothermia in infants is associated with increased mortality and morbidity. We noted consistent hypothermia postoperatively in more than 60% of our neonatal intensive care (NICU) babies. Therefore, we set out to determine whether a targeted quality improvement (QI) project could decrease postoperative hypothermia rates in infants. Our SMART aim was to reduce postoperative hypothermia (<36.5°C) in infants from 60% to 40% within 6 months. This project was approved by IRB at Guangzhou Women and Children's Medical Center, China. The QI team included multidisciplinary healthcare providers in China and QI experts from Children's Hospital of Philadelphia, USA. The plan-do-study-act (PDSA) cycles included establishing a perioperative-thermoregulation protocol, optimizing the transfer process, and staff education. The primary outcome and balancing measures were, respectively, postoperative hypothermia and hyperthermia (axillary temperature < 36.5°C, >37.5°C). Data collected was analyzed using control charts. The factors associated with a reduction in hypothermia were explored using regression analysis. There were 295 infants in the project. The percentage of postoperative hypothermia decreased from 60% to 37% over 26 weeks, a special cause variation below the mean on the statistical process control chart. Reduction in hypothermia was associated with an odds of 0.17 (95% CI: 0.06-0.46; p <.001) for compliance with the transport incubator and 0.24 (95% CI: 0.1-0.58; p =.002) for prewarming the OR ambient temperature to 26°C. Two infants had hyperthermia. Our QI project reduced postoperative hypothermia without incurring hyperthermia through multidisciplinary team collaboration with the guidance of QI experts fromthe USA.

Biochemical and molecular analysis of pediatric patients with metachromatic leukodystrophy in South China: functional characterization of five novel ARSA variants.

Metachromatic leukodystrophy (MLD) is a rare hereditary neurodegenerative disease caused by deficiency of the lysosomal enzyme arylsulfatase A (ARSA). This study described the clinical and molecular characteristics of 24 Chinese children with MLD and investigated functional characterization of five novel ARSA variants. A retrospective analysis was performed in 24 patients diagnosed with MLD at Guangzhou Women and Children's Medical Center in South China. Five novel mutations were further characterized by transient expression studies. We recruited 17 late-infantile, 3 early-juvenile, 4 late-juvenile MLD patients. In late-infantile patients, motor developmental delay and gait disturbance were the most frequent symptoms at onset. In juvenile patients, cognitive regression and gait disturbance were the most frequent chief complaints. Overall, 25 different ARSA mutations were identified with 5 novel mutations.The most frequent alleles were p.W320* and p.G449Rfs. The mutation p.W320*, p.Q155=, p.P91L, p.G156D, p.H208Mfs*46 and p.G449Rfs may link to late-infantile type. The novel missense mutations were predicted damaging in silico. The bioinformatic structural analysis of the novel missense mutations showed that these amino acid replacements would cause severe impairment of protein structure and function. In vitro functional analysis of the six mutants, showing a low ARSA enzyme activity, clearly demonstrated their pathogenic nature. The mutation p.D413N linked to R alleles. In western blotting analysis of the ARSA protein, the examined mutations retained reduced amounts of ARSA protein compared to the wild type. This study expands the spectrum of genotype of MLD. It helps to the future studies of genotype-phenotype correlations to estimate prognosis and develop new therapeutic approach.

Clinical analysis of 18 children with aggressive mature B-cell lymphoma after liver transplantation

Objective: To summarize the clinical characteristics, prognostic factors and treatment outcomes of childhood aggressive mature B-cell lymphoma after liver transplantation. Methods: This retrospective study included 18 children with newly diagnosed aggressive mature B-cell lymphoma after liver transplantation and treated from June 2018 to June 2022 in the Department of Hematology and Oncology of Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine. Clinical characteristics, treatment and outcomes of patients at last evaluation were analyzed. Overall survival (OS) and event free survival (EFS) rates were calculated by Kaplan-Meier method and Log-Rank analysis was performed to find factors of poor prognosis. Results: Among all 18 patients, there were 6 males and 12 females, and the age of onset was 40 (35, 54) months. The interval from transplant to tumor diagnosis was 21 (17, 35) months and 5 patients had early onset disease (<1 year since transplant). Seventeen patients had abdominal lesions. Diarrhea, vomiting and abdominal masses were the main clinical manifestations. All patients were Epstein-Barr virus (EBV) related posttransplant lymphoproliferative disorders (PTLD). One patient received individualized therapy due to critical sick at diagnosis, and the remaining 17 patients received CP (cyclophosphamide, methylprednisolone plus rituximab) and/or modified EPOCH (prednisone, etoposide, doxorubicin, vincristine, cyclophosphamide plus rituximab) regimens. Of all 18 patients, 15 cases got complete response, 2 cases got partial response, 1 patient died of severe infection. The 2-year OS and EFS rates of 18 patients were (94±5)% and (83±8)%, respectively. None of age, gender or early onset disease had effect on OS and EFS rates in univariate analysis (all P>0.05). Conclusions: The symptoms of PTLD were atypical. Close surveillance of EBV-DNA for patients after liver transplantation was crucial to early stage PTLD diagnosis. CP or modified EPOCH regimen was efficient for pediatric patients with aggressive mature B cell lymphoma after liver transplantation.

Treatment of ornithine transcarbamylase deficiency in a child with glyceryl phenylbutyrate

Glyceryl phenylbutyrate (GPB) serves as a long-term management medication for Ornithine transcarbamylase deficiency (OTCD), effectively controlling hyperammonemia, but there is a lack of experience in using this medicine in China. This article retrospectively analyzes the case of a child diagnosed with OTCD at Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, including a review of related literature. After diagnosis, the patient was treated with GPB, followed by efficacy follow-up and pharmacological monitoring. The 6-year and 6-month-old male patient exhibited poor speech development, disobedience, temper tantrums, and aggressive behavior. Blood ammonia levels peaked at 327 μmol/L; urine organic acid analysis indicated elevated uracil levels; cranial MRI showed extensive abnormal signals in both cerebral hemispheres. Genetic testing revealed de novo mutation in the OTC gene (c.241T>C, p.S81P). Blood ammonia levels were approximately 43, 80, and 56 μmol/L at 1, 2, and 3 months after starting GPB treatment, respectively. During treatment, blood ammonia was well-controlled without drug-related adverse effects. The patient showed improvement in developmental delays, obedience, temperament, and absence of aggressive behavior.

Intravenous Lacosamide Therapy for Pediatric Patients With Cluster Seizures

BackgroundFew studies have investigated intravenous lacosamide use to treat cluster seizures in pediatric patients. Therefore, we aimed to investigate the efficacy and safety of intravenous lacosamide therapy in pediatric patients with cluster seizures. MethodsWe retrospectively evaluated the efficacy and safety of intravenous lacosamide therapy in 25 pediatric patients with cluster seizures at Saitama Children's Medical Center between March 2019 and June 2023. Cluster seizures were defined as a single seizure of less than five minutes duration, repeated three or more times within 12 hours, with recovery of consciousness between seizures. Response was defined as seizure freedom for at least 12 hours after lacosamide infusion. ResultsThe median age at onset of epilepsy was 1.5 (0.0 to 9.8) years. The median seizure frequency was 5 (3 to 20) times per 12 hours. The etiologies were remote (n = 17), acute (n = 4), and progressive (n = 4). The median age at which intravenous lacosamide therapy was administered was 4.2 (0.0 to 11.3) years. The median lacosamide dose was 2.6 (1.3 to 5.2) mg/kg. In total, 12 of 25 patients (48.0%) responded. Among patients treated with intravenous lacosamide as first-line therapy, nine of 17 (52.9%) had complete seizure remission. The frequency of complete seizure remission in patients with remote etiologies was 58.8% (10 of 17); among them, seven of 12 (58.3%) patients with structural abnormalities showed complete seizure remission. No adverse events were observed. ConclusionsIntravenous lacosamide therapy is a potentially useful treatment option for cluster seizures in pediatric patients.

Prescribing Patterns of Nonrecommended Medications for Children With Acute COVID-19.

Repurposed medications for acute coronavirus disease 2019 (COVID-19) continued to be prescribed after results from rigorous studies and national guidelines discouraged use. We aimed to describe prescribing rates of nonrecommended medications for acute COVID-19 in children, associations with demographic factors, and provider type and specialty. In this retrospective cohort of children <18 years in a large United States all-payer claims database, we identified prescriptions within 2 weeks of an acute COVID-19 diagnosis. We calculated prescription rate, performed multivariable logistic regression to identify risk factors, and described prescriber type and specialty during nonrecommended periods defined by national guidelines. We identified 3 082 626 COVID-19 diagnoses in 2 949 118 children between March 7, 2020 and December 31, 2022. Hydroxychloroquine (HCQ) and ivermectin were prescribed in 0.03% and 0.14% of COVID-19 cases, respectively, during nonrecommended periods (after September 12, 2020 for HCQ and February 5, 2021 for ivermectin) with considerable variation by state. Prescription rates were 4 times the national average in Arkansas (HCQ) and Oklahoma (ivermectin). Older age, nonpublic insurance, and emergency department or urgent care visit were associated with increased risk of either prescription. Additionally, residence in nonurban and low-income areas was associated with ivermectin prescription. General practitioners had the highest rates of prescribing. Although nonrecommended medication prescription rates were low, the overall COVID-19 burden translated into high numbers of ineffective and potentially harmful prescriptions. Understanding overuse patterns can help mitigate downstream consequences of misinformation. Reaching providers and parents with clear evidence-based recommendations is crucial to children's health.

Changes in the epidemiology and clinical characteristics of viral gastroenteritis among hospitalized children in the Mainland of China: a retrospective study from 2016 to 2020

BackgroundAcute gastroenteritis (AGE) causes significant morbidity in children worldwide; however, the disease burden of children hospitalized with viral gastroenteritis in China has been rarely described. Through this study, we analyzed the data of hospitalized children with viral gastroenteritis to explore the changes in the epidemiology and clinical characteristics of viral gastroenteritis in the mainland of China.MethodsData were extracted from Futang Children's Medical Development Research Center (FRCPD), between 2016 and 2020, across 27 hospitals in 7 regions. The demographics, geographic distribution, pathogenic examination results, complications, hospital admission date, length of hospital stays, hospitalization charges and outcomes were collected and analyzed.ResultsViral etiological agents included rotavirus (RV), adenovirus (ADV), norovirus (NV) and coxsackievirus (CV) that were detected in 25,274 (89.6%), 1,047 (3.7%), 441 (1.5%) and 83 (0.3%) cases. There was a higher prevalence of RV and NV infection among children younger than 3 years of age. RV and NV had the highest detection rates in winter, while ADV in summer. Children with viral gastroenteritis were often accompanied by other diseases, such as myocardial diseases (10.98–31.04%), upper respiratory tract diseases (1.20–20.15%), and seizures (2.41–14.51%). Among those cases, the co-infection rate with other pathogens was 6.28%, with Mycoplasma pneumoniae (M. pneumoniae), Epstein-Barr virus (EBV), and influenza virus (FLU) being the most common pathogens. The median length of stay was 5 days, and the median cost of hospitalization corresponded to587 US dollars.ConclusionsThis finding suggests that viral gastroenteritis, especially those caused by RV, is a prevalent illness among younger children. Co-infections and the presence of other diseases are common. The seasonality and regional variation of viral etiological agents highlight the need for targeted prevention and control measures. Although viral gastroenteritis rarely leads to death, it also results in a significant economic burden on healthcare systems.

Sodium removal per ultrafiltration volume in automated peritoneal dialysis in pediatric patients.

The standard rate of sodium removal in adult anuric patients on continuous ambulatory peritoneal dialysis (CAPD) is 7.5g/L of ultrafiltration volume (UFV). Although automated PD (APD) is widely used in pediatric patients, no attempt has yet been made to estimate sodium removal in APD. The present, retrospective cohort study included pediatric patients with APD who were managed at Tokyo Metropolitan Children's Medical Center between July 2010 and November 2017. The patients underwent a peritoneal equilibrium test (PET) at our hospital. Sodium removal per UFV was calculated by peritoneal function and dwell time using samples from patients on APD with 1- and 2-h dwell effluent within three months of PET and 4- and 10-h dwell effluent at PET. In total, 217 samples from 18 patients were included, with 63, 81, and 73 of the samples corresponding to the High [H], High-average [HA], and Low-average [LA] PET category, respectively. Sodium removal per UFV (g/L in salt equivalent) for dwell times of one, two, four, and ten hours was 5.2, 8.8, 8.0, and 11.5 for PET [H], 5.3, 5.8, 5.6, and 8.1 for PET [HA], and 4.6, 5.1, 5.1, and 7.1 for PET [LA], respectively. Sodium removal per UFV in pediatric APD was less than the standard adult CAPD and tended to be lower with shorter dwell times, leading to sodium accumulation. Therefore, salt intake should be restricted in combination with one or more long daytime dwells, especially in anuric patients.

Spatial distribution of pediatric cancer patients using Geographic Information System (GIS) across the Philippines

Background: Every year, many children around the world get cancer, and this is a severe health problem. The Philippines is one such country where childhood cancer is a leading cause of death. This study uses unique maps (Geographic Information Systems or GIS) to examine these young cancer patients' locations. By doing this, we hope to find areas with more cases and see if children in those areas have easy access to the medical care they need. Methods: Methods involved primary and secondary data collection, including surveys and hospital records, with geospatial data analyzed using QGIS 3.4 Madeira. The study focuses on four Department of Health (DOH) tertiary hospitals: Philippine Children Medical Center (PCMC), Bicol Regional Training and Training Hospital (BRTTH), Southern Philippines Medical Center (SPMC), and Vicente Sotto Memorial Medical Center (VMMC) Participants from significant regions contributed to the study. Results: Results indicate that cancer centers, notably the primary children’s hospital, cater to patients from far-reaching areas, causing them to consult distant hospitals despite accessible alternatives. Nearly all (around 92.5%) patients seek care at specialized hospitals, even though only a small proportion (roughly 12.3%) live close to such facilities. Instead, 37.3% reported the closest health facility (within 10 kilometers) is a secondary government hospital, increasing the burden on patients with additional travel and non-medical costs. Conclusions: The study concludes that pediatric cancer patients face challenges accessing healthcare facilities, necessitating alternative methods like telemedicine and mobile clinics. The findings underscore the need for improved healthcare infrastructure and human resources to address the burdens faced by these patients.

Neurologic complications of thiamine (B1) deficiency following bariatric surgery in adolescents

BackgroundThe prevalence of obesity among children and adolescents is rising and poses a major health concern. Bariatric surgery is well established in adults and has become an option for adolescents. Thiamine (B1) deficiency is common following bariatric surgery in adults. It may present as Beri-Beri, Wernicke encephalopathy, or Korsakoff psychosis. ObjectiveOur aim was to describe the clinical features, diagnosis, and treatment of adolescents who presented with B1 deficiency after bariatric surgery at one center, and to summarize the data from the literature. PatientsThree adolescents with morbid obesity (two boys and one girl, aged 15.5 to- 17-years-old), presented at Schneider Children's Medical Center of Israel with progressive lower limb pain and weakness 2–3 month following a bariatric procedure (sleeve gastrectomy or narrowing of a bariatric band). The girl also had upper limb involvement and cerebellar signs. All three were non-compliant with micronutrient supplementation. After admission, they received intravenous B1 and oral multivitamin supplementation, and their symptoms improved considerably. ConclusionsMicronutrient supplementation following bariatric surgery is crucial to prevent deficiencies. In adolescents, compliance with micronutrient supplementation should be assessed before and after such surgery. Thiamine deficiency may cause polyneuropathy, among other symptoms. Treatment reduces the severity of neurological complications.

Prevalence of SARS-CoV-2 in newborns born to SARS-CoV-2-positive mothers at 2 weeks of life.

Limited evidence exists on management recommendations for neonates born to SARS-CoV-2-positive mothers. This study looked at transmission risk of neonates presenting for primary care in a large regional health system within New York during the early months of the COVID-19 pandemic. This was a prospective, observational study of newborns born to SARS-CoV-2-positive mothers presenting at any of the 19 Northwell Health-Cohen Children's Medical Center primary care practices who underwent another oropharyngeal/nasopharyngeal swab for detection of SARS-CoV-2 by day of life (DOL) 14. Among 293 newborns born to SARS-CoV-2-positive mothers who were negative at birth, 222 were retested at DOL 14, corresponding to times with different predominant strains. Of these, seven tested positive but had no symptoms. The overall low transmission rates and absence of symptomatic infection support the safety of direct breastfeeding after hospital discharge with appropriate hand and breast hygiene.

The Application of Umbilical Cord Blood-derived Platelet Gel for Skin Ulcers Associated With Chronic Graft-Versus-Host Disease in Pediatrics: A Randomized Trial

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a curative strategy against a variety of malignant and nonmalignant disorders. However, acute and chronic graft-versus-host disease (aGVHD and cGVHD, respectively) commonly complicate this approach, culminating in substantial morbidities and mortalities. The integumentary system is the preponderant organ involved in cGVHD, and its response to existing treatments, including well-versed immunosuppressants and novel targeted therapies, is not desirable. Despite the rarity, ulcers of sclerotic skin cGVHD are treatment-refractory and associated with significant morbidities and an exaggerated risk of infectious complications. Platelet-rich plasma (PRP) and its derivatives are endowed with growth factors and proangiogenic molecules and hold regenerative potential. This study aimed to assess the safety and efficacy of the application of platelet gel-containing dressing against ulcerative skin cGVHD in pediatric patients. This randomized trial is conducted at the hematopoietic stem cell transplantation unit of the Children's Medical Center Hospital in Tehran, Iran. Twenty-one pediatric patients (aged between 5 and 15 years) were initially enrolled, and 16 met the inclusion criteria. All cases (4 females) were recipients of allo-HSCT who had been complicated with symmetrically or near-symmetrically ulcerative sclerotic skin cGVHD. Fresh umbilical cord blood (UCB) was obtained from healthy donors and underwent centrifugation using a novel PRP preparation kit in a single-step process. Platelet gel was produced by adding thrombin to the isolated buffy coat layer. Two similar ulcers of each patient were randomized to receive either conventional dressing or platelet gels up to 6 times. At each time point evaluation, ulcer size and its relative reduction compared to the basal size were recorded. Included patients received a total of 80 platelet gel-containing dressings. While the mean sizes of randomized ulcers at the beginning of the study were similar, their differences became significant 15 days after the initiation of intervention (P = .019). In addition, the mean reduction in the ulcers’ surface area (in comparison to their baseline values) was significantly higher for the intervention arm at all evaluation points (P = .001 for day 5 and P < .001 for subsequent time points). At the end of the trial, the number of ulcers with a more than 50% reduction in size was 14 (87.5%) in the intervention arm (including 6 completely healed ulcers) versus 1 (6.25%, which was not completely healed) in the control arm (P < .001). None of the patients exhibited any localized or systemic treatment-related adverse events. In this study, using a relatively large number of cases, we showed that UCB-derived platelet gel is a safe, feasible, and effective curative approach for skin ulcers of sclerotic skin cGVHD in pediatric patients. Designing upcoming trials on the efficacy of this therapeutic approach for ocular, mucosal, and acute skin GVHD is prudent. Retrospectively registered at the Iranian Registry of Clinical Trials (registration number IRCT20190101042197N1) on August 24, 2020.