Treatment Outcomes In Children Research Articles

Results of treatment of children with complications after burns of the esophagus from button batteries

Aim. To enhance the treatment outcomes in children with complications following an esophageal burn caused by a disk battery.Materials and Methods. We analyzed the treatment outcomes of 102 patients suffering from esophageal burns from a disk battery, who were treated at the N.F. Filatov Children’s City Hospital of the Moscow Healthcare Ministry from 2009 to 2021. Children who developed complications (n=57) were divided according to the treatment strategy employed: conservative tactics (n=36, 63.2%) and radical surgery (n=21, 36.8%).Results and Discussion. The types of surgical interventions carried out for tracheoesophageal fistula (TEF) (n=31) included laparoscopic fundoplication and gastrostomy (38.7%), separation of TEF (41.9%), and tracheal plastic surgery with an esophageal flap and extirpation of the esophagus (19.4%). Assessment of the outcomes of TEF post laparoscopic fundoplication and gastrostomy demonstrated spontaneous closure in 63.6% of the cases. The average time until closure was 5 months. Among children with esophageal stenosis (n=24), esophageal bougienage was performed in 95.8% of the cases. All children identified with esophageal perforation (n=4) were successfully treated conservatively. Tactics for managing laryngeal paresis or paralysis included conservative therapy and observation in 33.3% of cases, tracheostomy in 29.2%, and lateralization procedures in 37.5%.Conclusion. Conservative tactics demonstrated superior results in all complications, minimizing the number of postoperative complications (16.7%) and ensuring favorable long-term outcomes.

Screening for parent and child ADHD in urban pediatric primary care: pilot implementation and stakeholder perspectives

BackgroundADHD commonly co-occurs in children and parents. When ADHD is untreated in parents, it contributes to negative child developmental and treatment outcomes. Screening for parent and child ADHD co-occurrence in pediatric primary care may be an effective strategy for early identification and treatment. There is no data on whether this screening model can be implemented successfully and there exists limited guidance on how to effectively approach parents about their own ADHD in pediatric settings. Even greater sensitivity may be required when engaging with families living in urban, low SES communities due to systemic inequities, mistrust, and stigma.MethodsThe current pilot study described the first 6 months of implementation of a parent and child ADHD screening protocol in urban pediatric primary care clinics serving a large population of families insured through Medicaid. Parents and children were screened for ADHD symptoms at annual well-child visits in pediatric primary care clinics as part of standard behavioral health screening. Independent stakeholder group meetings were held to gather feedback on factors influencing the implementation of the screening and treatment strategies. Mixed methods were used to examine initial screening completion rates and stakeholder perspectives (i.e., parents, primary care office staff, pediatricians, and behavioral health providers) on challenges of implementing the screening protocol within urban pediatric primary care.ResultsScreening completion rates were low (19.28%) during the initial 6-month implementation period. Thematic analysis of stakeholder meetings provided elaboration on the low screening completion rates. Identified themes included: 1) divergence between provider enthusiasm and parent hesitation; 2) parent preference versus logistic reality of providers; 3) centering the experiences of people with marginalized identities; and 4) sensitivity when discussing parent mental health and medication.ConclusionsFindings highlight the importance of developing flexible approaches to screening parent and child ADHD in urban pediatric health settings and emphasize the importance of cultural sensitivity when working with marginalized and under-resourced families.Trial registrationNCT04240756 (27/01/2020).

Recovery Rate and Treatment Outcome in Children Aged 6–59 Months with Severe Acute Malnutrition Admitted to Outpatient Therapeutic Feeding, in Ethiopia

ABSTRACT Different factors influence the recovery rate and treatment outcome of children with wasting using ready-to-use therapeutic food (OTP). Hence, our study aimed to assess determinants of the recovery rate among children with severe acute malnutrition (SAM) managed in OTP. A facility-based retrospective cohort study was conducted on 561 children treated in OTP. The proportion of recovery was 77.4%. The presence of edema, provision of amoxicillin, folic acid, and weight gain in the first 3 weeks were determinants of improved recovery rate. A better recovery rate could be achieved through improving the adherence to the proper administration of routine medications.

RARE-02. OBSERVATIONAL REGISTRY STUDY OF TREATMENT PRACTICES AND LONG-TERM OUTCOMES OF CHILDREN WITH NEUROFIBROMATOSIS TYPE 1 (NF1) AND PLEXIFORM NEUROFIBROMAS (PN) INITIATING SELUMETINIB IN REAL-WORLD PRACTICE IN THE UNITED STATES (US): STUDY DESIGN AND METHODOLOGY

Abstract Selumetinib (ARRY-142886, AZD6244) is an oral, allosteric MEK1/2 inhibitor approved in the US for children aged ≥2 years with NF1 and symptomatic, inoperable PN, based on results from the pivotal SPRINT study. Aims of the US Selumetinib Registry study [NCT05683678] include understanding treatment practices and assessing short- and long-term safety and effectiveness outcomes of selumetinib treatment in children with NF1-PN in real-world US practice. Clinical and non-clinical factors affecting outcomes will also be explored. This observational registry study of pediatric patients with NF1-PN will be conducted in up to 22 US centers. Eligible patients will be 2–18 years old at the time of selumetinib initiation (on/after April 10, 2020), and not currently participating in a clinical trial. Patients will be divided into three cohorts: Cohort 1 – treatment discontinued before enrollment, Cohort 2 – treatment initiated before enrollment and currently on treatment, Cohort 3 – treatment initiation intended within 3 months of enrollment. Patients will be followed for ≥36 and up to 60 months. Primary objectives are to describe patient demographics and disease characteristics (including diagnosis criteria and related manifestations, diagnostic tests and results, and PN-related morbidities), selumetinib treatment course, short- and long-term effectiveness and safety, and disease course and treatment following discontinuation. Key secondary objectives include measures of quality of life, pain and physical functioning before, during, and after selumetinib. Target enrollment is 200 patients with a 24-month enrollment period and will begin in 2023. The US Registry study will facilitate understanding of treatment practices and assess short and long-term outcomes of selumetinib for NF1-PN in a real-world setting.

Short-course, high-dose primaquine regimens for the treatment of liver-stage vivax malaria in children

ObjectivesTo assess the pharmacokinetics, safety, and tolerability of two high-dose, short-course primaquine (PQ) regimens compared with standard care in children with Plasmodium vivax infections. MethodsWe performed an open-label pediatric dose-escalation study in Madang, Papua New Guinea (Clinicaltrials.gov NCT02364583). Children aged 5-10 years with confirmed blood-stage vivax malaria and normal glucose-6-phosphate dehydrogenase activity were allocated to one of three PQ treatment regimens in a stepwise design (group A: 0.5 mg/kg once daily for 14 days, group B: 1 mg/kg once daily for 7 days, and group C: 1 mg/kg twice daily for 3.5-days). The study assessments were completed at each treatment time point and fortnightly for 2 months after PQ administration. ResultsBetween August 2013 and May 2018, 707 children were screened and 73 met the eligibility criteria (15, 40, and 16 allocated to groups A, B, and C, respectively). All children completed the study procedures. The three regimens were safe and generally well tolerated. The pharmacokinetic analysis indicated that an additional weight adjustment of the conventionally recommended milligram per kilogram PQ doses is not necessary to ensure the therapeutic plasma concentrations in pediatric patients. ConclusionsA novel, ultra-short 3.5-day PQ regimen has potential benefits for improving the treatment outcomes in children with vivax malaria that warrants further investigation in a large-scale clinical trial.

Rotator Cuff Injuries in the Pediatric Population: A Retrospective Review of Patient Characteristics and Treatment at a Single Center.

As youth participation in contact and overhead sports has increased in recent decades, so has the occurrence of injuries of the shoulder. Rotator cuff injury (RCI) is an infrequent shoulder pathology in pediatric patients and its description in the literature has been scarce. A greater understanding of RCI characteristics and treatment outcomes in children and adolescents would improve our understanding of this pathology and help to better guide clinical decision-making. To identify pediatric patients with magnetic resonance imaging-confirmed RCI treated at a single center to summarize injury characteristics, treatment, and outcomes. It was hypothesized that injuries would occur predominantly in overhead throwing athletes and would demonstrate good outcomes among both operatively and nonoperatively treated patients. Cross-sectional study. Level 4. A retrospective review of pediatric patients (<18 years old) diagnosed with and treated for an RCI between January 1, 2011 and January 31, 2021. Patient demographics, injury mechanism and type, treatment, and outcomes were collected. Descriptive statistics were performed. Bivariate testing was used to compare operatively and nonoperatively treated cohorts. A total of 52 pediatric patients treated for a rotator cuff avulsion, partial tear, or complete tear were identified. Mean age was 15 years and 67% of patients were male. Injuries were related most commonly to participation in throwing sports. Operative management occurred in 23% of patients, while 77% were managed nonoperatively. Treatment cohorts differed based on tear type, with all complete tears being managed operatively (P < 0.01). Associated shoulder pathology was common, with the most frequent finding being anterior shoulder instability pathology. Return to play was longer for operatively managed patients (7.1 vs 4.5 months; P < 0.01). The present study expands the limited data available regarding RCIs in pediatric patients. Most injuries are associated with sports and involve the supraspinatus tendon. RCIs were associated with good outcomes and low rates of reinjury in patients managed both nonoperative and operatively. RCI should be considered in throwing athletes with shoulder pain, even in skeletally immature patients. This retrospective study fills the hole in the literature by detailing the patterns associated with RCI characteristics and treatment outcomes. In contrast to studies of adult RCIs, our results suggest that outcomes are good regardless of treatment type.

Ethnic differences in treatment outcome for children and young people accessing mental health support

Children and Young People (CYP) from minoritized ethnic backgrounds experience structural inequalities in Children and Young People’s Mental Health Settings (CYPMHS). This mixed methods study explores whether CYP’s ethnicity is associated with their treatment outcomes (operationalised as ‘measurable change’) from CYPMHS. A multilevel multi-nominal regression analysis, controlling for age, gender, referral source, presenting difficulty, case closure reason, suggests that CYP from Asian backgrounds (OR = 0.82, CI [0.70, 0.96]) and Mixed-race (odds ratio (OR) = 0.80; 95% CI [0.69, 0.92]) are less likely to report measurable improvement in mental health difficulties compared to White British CYP. Three themes from a thematic analysis of semi-structured interviews with 15 CYP from minoritized ethnic backgrounds focused on views and experiences of ending mental health support are also presented. CYP view personalised support and the right therapist as conducive to good endings and valued a range of outcomes pertaining to empowerment. Experiences of stigma and inequalities may begin to explain the less positive outcomes experienced by Asian and Mixed-race CYP found in the regression analysis. The implications of these findings and future areas of research are suggested.

Treatment of pediatric relapsed and refractory acute myeloid leukemia

Background. Despite the modern therapy programs including hematopoietic stem cell transplantation, the treatment outcomes for children with acute myeloid leukemia (AML) remain unsatisfactory. The 5‑year overall survival rate is about 70 %. The 5‑year overall survival rate for patients with relapsed and refractory AML is 2 times lower (about 35 %). The treatment failure rate in primary AML and unsatisfactory results in relapsed and refractory AML make it necessary to optimize therapy protocols.Aim was a long-term retro- and prospective analysis of clinical and laboratory characteristics and treatment outcomes in patients with relapsed and refractory forms of AML.Materials and methods. This article presents the treatment results of 54 patients from 1 to 18 years of age, with relapsed and refractory AML treated at the N. N . Blokhin National Medical Research Center of Oncology from 1997 to 2022.Results. A comparison of 5 different programs revealed that patients who received second remission induction with the FLA + FLA scheme had 81.8 % of response (complete or partial) achievement. Analysis of the results in achievement the second remission in patients received epigenetic agents (azacytidine, decitabine, valproic and all-trans retinoid acids) with second-line chemotherapy found that treatment response rate was 100 % (n = 27), in contrast to patients received only second-line chemotherapy (n = 27) – 81.5 % (p = 0.003). The best treatment results were in group of patients whose treatment included epigenetic agents and allogenic hematopoietic stem cell transplantation after second remission induction – 5‑year overall survival was 51.3 ± 9.7 %.Conclusion. Intensive polychemotherapy with fludarabine- and cytarabine-containing regiments with following allogeneic hematopoietic stem cell transplantation and epigenetic agents are current trend and pathogenetically based approach for relapsed and refractory pediatric AML. Probable, the definition of the role and place of targeted drugs (gemtuzumab ozogamicin) could continue the advances in treatment of such unfavorable patient group.

Dose frequency randomized controlled trial for Dynamic Temporal and Tactile Cueing (DTTC) treatment for childhood apraxia of speech: protocol paper

BackgroundChildhood apraxia of speech (CAS) is a pediatric motor-based speech sound disorder that requires a specialized approach to intervention. The extant literature on the treatment of CAS commonly recommends intensive treatment using a motor-based approach, with some of the best evidence supporting the use of Dynamic Temporal and Tactile Cueing (DTTC). To date, a rigorous and systematic comparison of high and low dose frequency (i.e., frequency of therapy sessions) has not been undertaken for DTTC, resulting in a lack of evidence to guide decisions about the optimal treatment schedule for this intervention. The current study aims to fill this gap in knowledge by comparing treatment outcomes when dose frequency is varied.MethodsA randomized controlled trial will be conducted to examine the efficacy of low versus high dose frequency on DTTC treatment outcomes in children with CAS. A target of 60 children, 2;6–7;11 years of age, will be recruited to participate in this study. Treatment will be provided in the community setting by speech-language pathologists who have completed specialized training administering DTTC in a research reliable manner. True randomization with concealed allocation will be used to assign children to either the low or high dose frequency group. Treatment will be administered in 1-h sessions either 4 times per week over a 6-week period (high dose) or 2 times per week over a 12-week period (low dose). To measure treatment gains, probe data will be collected before treatment, during treatment, and 1 day, 1 week, 4 weeks, and 12 weeks post-treatment. Probe data will consist of customized treated words and a standard set of untreated words to assess generalization of treatment gains. The primary outcome variable will be whole word accuracy, encompassing segmental, phonotactic, and suprasegmental accuracy.DiscussionThis will be the first randomized controlled trial to evaluate dose frequency for DTTC treatment in children with CAS.Trial registrationClinicalTrials.gov identifier NCT05675306, January 6, 2023.

Clinical spectrum of contactin-associated protein 2 autoimmune encephalitis in children.

Anti-contactin-associated protein 2 (CASPR2)-related autoimmune encephalitis (AE) is more common in adults than in children. Clinical understanding of anti-CASPR2-antibody (Ab)-related AE, diagnosis and treatment standards are lacking in children. Therefore, this retrospective study on clinical symptoms and treatment outcomes in children with anti-CASPR2-Ab-related AE was conducted, to improve the clinical understanding of the disease, its diagnosis and treatment. This study retrospectively assessed children with anti-CASPR2-Ab-related AE from January 1, 2020, to June 30, 2022, in the Department of Neurology at Hunan Children's Hospital. Data regarding demographics, clinical symptoms, laboratory examinations, electroencephalography (EEG), imaging, and curative were collected. Thirteen patients were positive for serum anti-CASPR2-Ab (age at manifestation, 25 months to 13 years old; median, 8.1 years old; male-to-female ratio, 8/5). One patient (P1) had dual Abs, including anti-CASPR2 and anti-N-methyl-D-aspartate receptor Abs; his symptoms were more severe than those of children with anti-CASPR2 Abs alone. The clinical symptoms of the 13 patients with anti-CASPR2 Ab were movement disorders (9/13), consciousness disorders (9/13), abnormal demeanor (8/13), seizures (7/13), language disorders (6/13), fever (6/13), pain (4/13), involuntary exercise (4/13), poor diet (4/13), vomiting (3/13), sleep disorders (3/13), mood disorders (3/13), eczema/itching/redness (2/13), sweating (P8), urinary disorders (P13), and cognitive disorders (P9). No tumors were found in any patient. Additionally, EEG results of six patients were abnormal and imaging findings such as abnormal signals were found in 10 patients. Moreover, all except one patient recovered well after treatment; P1 with overlapping syndrome underwent recovery for more than 2 years. None of the patients who recovered have had a relapse. Anti-CASPR2-Ab-related AE has several clinical manifestations. Anti-CASPR2-Ab levels were higher in male patients than in female patients. Moreover, related tumors are relatively rare. Most patients benefit from immunotherapy and have a lower chance of recurrence in the short term. Furthermore, different from patients who had anti-CASPR2-Ab AE alone, those with overlapping syndrome had a severe and complex condition requiring lengthy treatment and rehabilitation. Additional studies are needed to evaluate the long-term prognosis of these patients.

The relation of changes in intraoperative EEg and ECog with age, anamnesis duration and outcome of surgical treatment in children with symptomatic epilepsy

Aim. To reveal relation of change in intraoperative electroencephalography (EEG), electrocorticography (EсoG) with age of patients, the anamnesis duration, localization and type of pathology, the result of treatment. Materials and methods. 64 patients with symptomatic epilepsy operated at N.N. Burdenko National Medical Research Center of Neurosurgery of the Ministry of Health of Russia from 2010 to 2016. 28 males, 36 females, age from 3 months to 18 years, on average 7,2 years. The pathology localization: temporal – 21 cases, occipital and parietal – 18, frontal – 25. Pathomorphology: 12 patients with focal cortical dysplasia (FCD) I, 20 patients with FCD II, 5 patients with FCD III, 4 patients with tuberous sclerosis, 20 patients with low grade tumors, and 3 cases else. The duration of diseaseis from 61 to 5081 days. Catamnesis (follow-up) ranged from 364 to 1877 days. There was four criteria for including patients in research: intraoperative EEG, presence of initial and control registration of EсoG, follow-up above 364 days. There are three hallmarks selected as significant concerning the efficiency of neurophysiological monitoring: 1) the positive changes in scalp EEG: decrease of interhemispheric asymmetry due to reduction of slow activity on the side of pathology; significant reduction of the unilateral epileptiform activity on the side of pathology and bilateral if exist; 2) the positive changes in EсoG: significant reduction of the regular epileptiform activity; 3) the absence or existence of residual epileptiform activity in adjacent to resected pathology sites. The statistical processing carried out with application of a Stat10 package. Results. The more expressed positive dynamics of EEG were revealed in patients of smaller age already during operation. A correlation was found between the age of patients and the positive dynamics of intraoperative EEG. An inverse correlation was found between the duration of the disease and the positive dynamics of the scalp EEG during surgery (p = 0.1560). The EEG changes during surgery were shown more often with reduction of the seizure anamnesis. There was found out that residual epileptiform activity on ECoG occurred more often at patients with longer anamnesis of a disease and at patients with frontal localization of pathology. The residual epileptiform activity on ECoG was registered more often at patients with preservation of seizures (outcomes 2, 3, 4, 5 ILAE). Conclusions. During resective interventions for symptomatic epilepsy in children the probability of positive changes was the more, the younger the patient was. Intraoperative ECoG is one of the useful tools in epilepsy surgery, but it does not definitely determine success. There was no statistically significant relationship between the presence of residual epileptiform signs on the ECoG in the cortex adjacent to the removal area and the outcome of surgical treatment for seizures. Patients in follow-up had three times fewer seizures in the absence of residual epileptiform activity on the ECoG of adjacent to the resected areas of the cortex. The dependence is statistically insignificant. Removal of FCD type III and tumors is accompanied by a more complete elimination of epileptiform activity than FCD I, the level of statistical significance of p = 0.0305.

Long-Term Outcomes of Antegrade Continence Enemas to Treat Constipation and Fecal Incontinence in Children.

The objective of this study is to investigate long-term outcomes of antegrade continence enema (ACE) treatment in children with constipation or fecal incontinence. Prospective cohort study including pediatric patients with organic or functional defecation disorders who started ACE treatment. Data were collected at baseline and at follow-up (FU) from 6 weeks until 60 months. We assessed parent and patient-reported gastrointestinal health-related quality of life (HRQoL) using the Pediatric Quality of Life Inventory Gastrointestinal Symptoms Module (PedsQL-GI), gastrointestinal symptoms, adverse events, and patient satisfaction. Thirty-eight children were included (61% male, median age 7.7 years, interquartile range 5.5-12.2). Twenty-two children (58%) were diagnosed with functional constipation (FC), 10 (26%) with an anorectal malformation, and 6 (16%) with Hirschsprung disease. FU questionnaires were completed by 22 children (58%) at 6 months, 16 children (42%) at 12 months, 20 children (53%) at 24 months, and 10 children (26%) at 36 months. PedsQL-GI scores improved overall with a significant increase at 12- and 24-month FU for children with FC and a significant increase in parent reported PedsQL-GI score at 36-month FU for children with organic causes. Minor adverse events, such as granulation tissue, were reported in one-third of children, and 10% of children needed a surgical revision of their ACE. The majority of all parents and children reported that they would "probably" or "definitely" choose ACE again. ACE treatment is perceived positively by patients and parents and can lead to long-term improvement in gastrointestinal HRQoL in children with organic or functional defecation disorders.

Pediatric paragonimiasis: a retrospective analysis of cases from a county in south-west China.

The clinical manifestations of paragonimiasis are diverse and non-specific, and can easily lead to misdiagnosis. We aimed to analyze the clinical manifestations, laboratory features, treatment, and clinical outcome of children with paragonimiasis in order to improve recognition of this disease and avoid misdiagnosis. Children diagnosed with paragonimiasis from August 2016 to July 2022 were included in the study. Information on population informatics, medical history, and laboratory features was extracted from case data. The clinical features of paragonimiasis were retrospectively analyzed. A total of 45 children were included in this study. All children had, at least, one risk factor. The clinical features mainly included fever, cough, pleural effusion, peritoneal effusion, and subcutaneous nodules. The main imaging findings were alveolar exudation, peritoneal effusion, pleural thickening, and local nodules. The "tunnel sign" finding on computed tomography (CT)/magnetic resonance imaging (MRI) was helpful in establishing the diagnosis of paragonimiasis. After praziquantel treatment, most of the children improved, and one child with cerebral paragonimiasis experienced sequelae. Most children with paragonimiasis have a good prognosis, but few children can experience sequelae. Avoidance of untreated water and raw food is a simple, feasible, and effective preventive measure.

A multicentre, retrospective audit of fosfomycin use for urinary tract infections in Australian children and adolescents.

Urinary tract infections (UTIs) due to MDR organisms are increasingly common. The lack of paediatric data on efficacious antibiotics makes UTI treatment particularly challenging. Data on the efficacy of fosfomycin use for UTI in children are variable. We conducted a retrospective audit of children aged 0-18 years who were treated with fosfomycin for UTI at seven tertiary paediatric hospitals in Australia over a 7 year period, from 2014 to 2020. Ninety-one children with a median age of 5 years (range 2 months to 18 years) received oral fosfomycin for UTI. The majority (57/91, 63%) had one or more comorbidity, with the most common being renal tract anomalies (24/91, 26%). Fifty-nine (65%) had febrile UTI, 14/91 (15%) had pyelonephritis and 1/91 (1%) was bacteraemic. A majority (80/91, 88%) of urinary cultures had an ESBL-producing Gram-negative pathogen isolated. Fosfomycin susceptibility was evident in all 80 isolates tested. For uncomplicated UTI, the most common dose in children aged <1, 1-12 and >12 years was 1, 2 and 3 g, respectively. For complicated UTI, doses of 2 and 3 g were most common. The median duration of fosfomycin administration was 5 days (range 1-82). Clinical cure was achieved in 84/90 (93%); the six with treatment failure had underlying comorbidities. Overall, 2/91 (2%) children experienced drug-related adverse effects comprising gastrointestinal symptoms in both, which resolved after treatment discontinuation. Fosfomycin is well tolerated and associated with favourable treatment outcomes in children with UTI. Further research on the optimal dosing strategy is required.

Olverembatinib Treatment in Pediatric Patients With Relapsed Philadelphia-Chromosome-Positive Acute Lymphoblastic Leukemia

Treatment outcomes for children with Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) remained poor despite the use of intensive chemotherapy, imatinib or dasatinib, and consolidative allogeneic hematopoietic cell transplantation. Oleverembatinib, a third-generation ABL inhibitor, was found to be highly effective and safe in adults with chronic myeloid leukemia and in some adults with relapsed or refractory Ph+ ALL. We reviewed the efficacy and safety profile of olverembatinib treatment in 6 children with relapsed Ph+ ALL and 1 with T-ALL and ABL class fusion, all of whom had previously received dasatinib or intolerance to dasatinib. The median duration of olverembatinib treatment was 70 days (range: 4-340) and the median cumulative dose was 600 mg (range: 80-3810). Complete remission with negative minimal residual level (<0.01%) was achieved in 4 of the 5 evaluable patients, 2 of whom were treated with olvermbatinib as a single agent. Safety profile in 6 evaluable patients was excellent with grade 2 extremity pain occurred in 2 patients and grade 2 myopathy of lower extremity and grade 3 fever in 1 patient each. Olverembatinib appeared to be safe and effective in children with relapsed Ph+ ALL.

Percutaneous approach to residual pulmonary bifurcation stenosis in conotruncal diseases.

Residual stenosis after right ventricle outflow tract surgery represents a major issue to manage in the children and adult patient with conotruncal defects. Despite a detailed multimodality imaging, the anatomy of distal pulmonary trunk and pulmonary artery bifurcation may be challenging in these patients.The aim of this study was to analyse retrospectively the outcome of the percutaneous transcatheter treatment in children with post-surgical stenosis of pulmonary artery bifurcation.We enrolled 39 patients with a median age of 6.0 years. Standard high-pressure balloon dilation was attempted in 33 patients, effective in 5 of them. Pulmonary branch stenting was performed in 10 patients, effective in 6. A kissing balloon approach was chosen in 17 patients (6 after angioplasty or stenting failure), and this technique was effective in 16 cases. Finally, a bifurcation stenting was performed in 10 patients (second step in 9 cases), effective in all the cases. None of the patients approached by kissing balloon needed a bifurcation stenting.In conclusion, standard balloon angioplasty and standard stenting might be ineffective in post-surgical stenosis involving pulmonary artery bifurcation. In this population, kissing balloon or bifurcation stenting, followed by side branch de-jailing, may be more effective in relieving the gradient.

Clinical Characteristics and Treatment Outcomes of Children with Primary Osteoporosis.

Primary osteoporosis is a rare and essential problem in childhood that can cause severe skeletal deformities. We aimed to reveal the spectrum of primary osteoporosis and assess the effectiveness and safety of bisphosphonates in increasing bone mineral density and reducing fractures. Patients with primary osteoporosis who received at least one course of pamidronate or zoledronic acid were included in the study. Patients were divided into 2 groups, osteogenesis imperfecta and non-osteogenesis imperfecta subjects. We evaluated bone densitometer parameters, activation scores, pain status, deformity status, and the number of fractures per year in all patients. Of the 31 patients, 21 with osteogenesis imperfect, 3 patients with spondyloocular syndromes, 2 with Bruck Syndrome, and 5 with idiopathic juvenile osteoporosis were included. A total of 21 patients had received pamidronate treatment, while only 4 received zoledronic acid, and 6 of them switched from pamidronate to zoledronic acid. At the end of the treatment, the mean bone mineral density height-adjusted Z-score increased from -3.39 ± 1.30 to -0.95 ± 1.34. The number of fractures per year decreased from 2.28 ± 2.67 to 0.29 ± 0.69. The activation score increased from 2.81 ± 1.47 to 3.16 ± 1.48. The pain decreased significantly. There was no difference in bone mineral density increase in patients treated with pamidronate or zoledronic acid. Those with osteogenesis imperfecta were diagnosed at an earlier age with severe deformity and fractures. Pamidronate and zoledronic acid increased bone mineral density in all types of primary osteoporosis.

Phase 2 study of combination chemotherapy with bortezomib in children with relapsed and refractory acute lymphoblastic leukemia.

Treatment outcomes for children with relapsed and refractory acute lymphoblastic leukemia (R/R-ALL) remain poor, and the optimal induction therapy has not been determined. Bortezomib is a proteasome inhibitor that acts synergistically and additively with standard chemotherapy for ALL. We evaluated the efficacy and safety of combination chemotherapy with bortezomib in children with R/R-ALL. This single-arm, multicenter, phase 2 study was conducted in Japan between 2016 and 2020. Eligible patients were divided into two cohorts: a high-risk first-relapse cohort of untreated patients with high-risk first-relapsed ALL and an expansion cohort of patients with refractory ALL, including multiple relapses, relapse after allogeneic hematopoietic cell transplantation, and induction failure. All patients received a single course of chemotherapy as induction therapy. Sixteen patients (10 in the high-risk first-relapse cohort, six in the expansion cohort) were evaluable. The overall remission rate after induction therapy was 60% in the high-risk first-relapse cohort and 16.7% in the expansion cohort. All patients had minimal residual disease. Adverse events were acceptable except for interstitial lung disease and hypoxia in a patient in the expansion cohort, but addition of bortezomib to conventional chemotherapy did not produce obvious improvement in children with R/R-ALL.

Treatment results of children with the diagnosis of acute myeloid leukemia. Is trisomy 21 an important cause of mortality?

Background: Acute myeloid leukemia (AML) is the second most common leukemia accounts for 20% of childhood leukemia. Intensive chemotherapy and hematopoietic stem cell transplantation (HSCT) improve outcomes. In this study, it was aimed to present the clinical features and treatment results of patients diagnosed with pediatric AML in the last three years. Methods: It is an observational clinical study, patients who received chemotherapy and HSCT with the diagnosis of AML between 2018 and 2020 were retrospectively evaluated. Age, gender, AML subtype, genetic characteristics, laboratory findings, treatment responses, febrile neutropenia and fungal infection frequencies of the patients were evaluated. HSCT indications, pre-transplant bone marrow aspiration results, transplant type, donor characteristics, and post-transplant complications of patients who underwent HSCT were recorded. Post-treatment status and treatment success rates of the patients were evaluated. Results: Of the 46 patients included in the study, 26 were female and 20 were male. The mean age was 8.2 ± 5.75 years. The most common subtypes were AML M4 and M5 with 9 patients. Total of 201 febrile neutropenia attacks were observed. Suspicious or definite proven Aspergillus infection was detected in 20 of the patients. HSCT was applied to 27 of the patients. Four patients were Down syndrome-myeloid leukemia (DS-ML) and FLT3-ITD mutation was detecetd in 5 patients. Conclusions: FLT3-ITD mutation, relapse disease, and pre-HSCT M2 bone marrow were found to be highly correlated with progressive disease and mortality. In addition, patients with Down syndrome had a high mortality due to additional comorbidity and drug adverse effects.

HIV matters when diagnosing TB in young children: an ancillary analysis in children enrolled in the INPUT stepped wedge cluster randomized study

BackgroundChildren under age five years, particularly those living with HIV (CLHIV), are at risk for rapid progression of tuberculosis (TB). We aimed to describe TB clinical presentations, diagnostic pathways and treatment outcomes in CLHIV compared to children without HIV in Cameroon and Kenya.MethodsThis sub-analysis of a cluster-randomized trial evaluating the integration of pediatric TB services from May 2019 to March 2021 enrolled children age < 5 years with TB. We estimated the HIV infection rate with 95% confidence interval (CI). We compared TB clinical presentations, diagnostic pathways and treatment outcomes in CLHIV and children without HIV. Finally, we investigated whether HIV infection was associated with a shorter time to TB diagnosis (≤ 3 months from symptoms onset) after adjusting for covariates. Univariable and multivariable logistic regression analysis were performed with adjusted odds ratios (AORs) presented as measures of the association of covariates with HIV status and with shorter time to TB diagnosis.ResultsWe enrolled 157 children with TB (mean age was 1.5 years) and 22/157 (14.0% [9.0-20.4%]) were co-infected with HIV. CLHIV were more likely to initially present with acute malnutrition (AOR 3.16 [1.14–8.71], p = 0.027). Most TB diagnoses (140/157, 89%) were made clinically with pulmonary TB being the most common presentation; however, there was weak evidence of more frequent bacteriologic confirmation of TB in CLHIV, 18% vs. 9% (p = 0.067), due to the contribution of lateral-flow urine lipoarabinomannan to the diagnosis. HIV positivity (AOR: 6.10 [1.32–28.17], p = 0.021) was independently associated with a shorter time to TB diagnosis as well as fatigue (AOR: 6.58 [2.28–18.96], p = 0.0005), and existence of a household contact diagnosed with TB (AOR: 5.60 [1.58–19.83], p = 0.0075), whereas older age (AOR: 0.35 [0.15–0.85], p = 0.020 for age 2–5 years), night sweats (AOR: 0.24 [0.10–0.60], p = 0.0022) and acute malnutrition (AOR: 0.36 [0.14–0.92], p = 0.034) were associated with a delayed diagnosis. The case fatality rate was 9% (2/22) in CLHIV and 4% (6/135) in children without HIV, p = 0.31.ConclusionsThese results altogether advocate for better integration of TB services into all pediatric entry points with a special focus on nutrition services, and illustrate the importance of non-sputum-based TB diagnostics especially in CLHIV.Trial registrationNCT03862261, first registration 05/03/2019.