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X-linked hypophosphatemia (XLH) is a genetic disease that results in excessive FGF23, chronic hypophosphatemia, and musculoskeletal abnormalities, with affected patients experiencing symptoms such as bone pain, bone deformity, fracture, and pseudofracture. Burosumab is a fully human monoclonal antibody that binds to FGF23, improving lowered serum 1,25(OH)2D and phosphate levels in patients with XLH. There are insufficient data on the use of burosumab, its safety, and the outcomes of treated patients in a real-world setting. The SUNFLOWER (Study of longitUdinal observatioN For patients with X-Linked hypOphosphatemic rickets/osteomalacia in collaboration With Asian partnERs) study is an ongoing longitudinal, observational cohort study of patients with XLH in Japan and South Korea. Enrollment occurred between April 2018 and December 2020. This interim analysis compared the background characteristics of patients who received burosumab with those who did not, and assessed improvements in biomarkers, physical and motor function, health-related quality-of-life (HRQOL) and other patient-reported outcome (PRO) measures, as well as the safety of burosumab treatment in 143 Japanese patients from 15 institutions over 6 mo. The patients had a median [interquartile range] age of 17.5 [11.0, 38.8] yr and 98 (68.5%) were female. Among patients aged <18 and ≥18 yr, 40/73 (54.8%) and 25/70 (35.7%) received burosumab, respectively. More patients aged ≥18 who received burosumab had bone pain at baseline vs those not treated with burosumab (6/25, 24.0% vs 2/45, 4.4%, p=.021). Patients treated with burosumab had improved serum phosphate and 1,25(OH)2D levels; moreover, rickets severity and HRQOL/PRO measures, such as pain, appeared to improve over 6 mo of burosumab treatment, and no new safety concerns were identified. This study identified trends in the background characteristics of patients with XLH who receive burosumab in real-world clinical practice. Furthermore, the results support the use of burosumab therapy in real-world settings.
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It has been postulated from a combination of evidence that a sudden increase in COVID-19 cases among pediatric patients after onset of the Omicron wave was attributed to a reduced requirement for TMPRSS2-mediated entry in pediatric airways with lower expression levels of TMPRSS2. Epidemic strains were isolated from the indigenous population in an area, and the levels of TMPRSS2 required for Delta and Omicron variants were assessed. As a result, Delta variants proliferated fully in cultures of TMPRSS2-positive Vero cells but not in TMPRSS2-negative Vero cell culture (350-fold, Delta vs 9.6-fold, Omicron). There was no obvious age-dependent selection of Omicron strains affected by the TMPRSS2 (9.6-fold, Adults vs. 12-fold, Children). A phylogenetic tree was generated and Blast searches (up to 100 references) for the spread of strains in the study area showed that each strain had almost identical homology (>99.5%) with foreign isolates, although indigenous strains had obvious differences from each other. This suggested that the differences had been present abroad for a long period. Therefore, the lower requirement for TMPRSS2 by Omicron strains might be applicable to epidemic strains globally. In conclusion, the property of TMPRSS2-independent cleavage makes Omicron proliferate with ease and allows epidemics among children with fewer TMPRSS2 on epithelial surfaces of the respiratory organs.
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COVID-19 , SARS-CoV-2 , Serina Endopeptidasas , SARS-CoV-2/genética , SARS-CoV-2/aislamiento & purificación , Humanos , Serina Endopeptidasas/genética , Serina Endopeptidasas/metabolismo , Chlorocebus aethiops , COVID-19/virología , COVID-19/metabolismo , COVID-19/epidemiología , Células Vero , Niño , Animales , Preescolar , Filogenia , Adulto , Lactante , Masculino , FemeninoRESUMEN
Short stature with IGF-1 receptor (IGF1R) gene alteration is known as small-for-gestational-age (SGA) short stature with elevated serum IGF1 levels. Its prevalence and clinical characteristics remain unclear. No adapted treatment is available for short stature related to IGF1R gene alteration in Japan, and genetic testing is not yet widely accessible. We investigated short stature with IGF1R gene alterations and analyzed the clinical data of 13 patients using the results of questionnaires issued to the Japanese Society for Pediatric Endocrinology. Four cases were caused by a deletion of chromosome 15q26.3, and eight were caused by heterozygous pathogenic variants in the IGF1R gene. Cases with deletions showed a more severe degree of growth impairment (-4.5 ± 0.43 SD) than those caused by pathological variants (-2.71 ± 0.15 SD) and were accompanied by neurodevelopmental delay. However, cases caused by pathological variants lacked distinctive features. Only three of the 12 cases demonstrated serum IGF1 values exceeding +2 SD, and the other three had values below 0 SD. Four patients did not meet the criteria for SGA at birth. Six patients received GH therapy for SGA short stature and showed improvement in growth rate without any side effects or elevated serum IGF1 levels during treatment. Elevated IGF1 levels (over +2 SD) after GH treatment should be considered a suspicious finding. Owing to the lack of distinctive features, there was a possibility of undiagnosed cases of this condition. Promoting genetic testing and clinical trials on GH administration for this condition is recommended.
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Trastornos del Crecimiento , Hormona de Crecimiento Humana , Recién Nacido Pequeño para la Edad Gestacional , Receptor IGF Tipo 1 , Humanos , Receptor IGF Tipo 1/genética , Femenino , Masculino , Niño , Hormona de Crecimiento Humana/uso terapéutico , Trastornos del Crecimiento/tratamiento farmacológico , Trastornos del Crecimiento/genética , Preescolar , Recién Nacido Pequeño para la Edad Gestacional/crecimiento & desarrollo , Factor I del Crecimiento Similar a la Insulina/metabolismo , Adolescente , Enanismo/tratamiento farmacológico , Enanismo/genética , Japón , Estatura/efectos de los fármacos , Resultado del TratamientoRESUMEN
In a randomized, open-label phase 3 study of 61 children aged 1-12 years old with X-linked hypophosphatemia (XLH) previously treated with conventional therapy, changing to burosumab every 2 weeks (Q2W) for 64 weeks improved the phosphate metabolism, radiographic rickets, and growth compared with conventional therapy. In this open-label extension period (weeks 64-88), 21 children continued burosumab Q2W at the previous dose or crossed over from conventional therapy to burosumab starting at 0.8 mg/kg Q2W with continued clinical radiographic assessments through week 88. Efficacy endpoints and safety observations were summarized descriptively for both groups (burosumab continuation, n = 6; crossover, n = 15). At week 88 compared with baseline, improvements in the following outcomes were observed in the burosumab continuation and crossover groups, respectively: mean (SD) RGI-C rickets total score (primary outcome), +2.11 (0.27) and +1.89 (0.35); mean (SD) RGI-C lower limb deformity score, +1.61 (0.91) and +0.73 (0.82); and mean (SD) height Z-score + 0.41 (0.50) and +0.08 (0.34). Phosphate metabolism normalized rapidly in the crossover group and persisted in the continuation group. Mean (SD) serum alkaline phosphatase decreased from 169% (43%) of the upper limit of normal (ULN) at baseline to 126% (51%) at week 88 in the continuation group and from 157% (33%) of the ULN at baseline to 111% (23%) at week 88 in the crossover group. During the extension period, treatment-emergent adverse events (AEs) were reported in all 6 children in the burosumab continuation group and 14/15 children in the crossover group. The AE profiles in the randomized and extension periods were similar, with no new safety signals identified. Improvements from baseline in radiographic rickets continued in the extension period among children with XLH who remained on burosumab. Children who crossed over from conventional therapy to burosumab demonstrated a rapid improvement in phosphate metabolism and improved rickets healing over the ensuing 22 weeks.
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Background: Elective cesarean sections (ECSs) for early-term pregnancies at 37 weeks of gestational age (GA) aim to reduce the risk of emergency cesarean sections due to the onset of labor or rupture of membranes. However, resultant increases in neonatal respiratory disorders, including transient tachypnea of the newborn (TTN) have been observed. However, few studies have elucidated the associated risk factors. Consequently, we aimed to determine whether differences existed in the clinical outcomes between neonates delivered via ECS at 37 weeks and those delivered at ≥ 38 weeks of GA. Methods: A retrospective analysis was conducted on 259 neonates born via ECS at Tottori University Hospital, between January 2013 and December 2019, with birthweights ≥ 2500 g and GAs > 37 weeks. The neonates were categorized into two cohorts: births at 37 and at ≥ 38 weeks of GA (37-week and 38-week cohorts). The principal clinical outcomes included the appearance, pulse, grimace, activity, and respiration (Apgar) scores, need for positive-pressure ventilation, incidence of TTN, and length of hospital stay. Results: No statistically significant differences were observed in the indications for ECS, sex, or birthweight between the two cohorts. The 37-week cohort exhibited a lower 1-min Apgar score than did the 38-week cohort, with no statistically significant differences between the two cohorts, at 5 min. Statistically significant differences were not observed in the need for positive-pressure ventilation during initial resuscitation or length of hospital stay for patients with TTN between the two cohorts. Notably, the 37-week cohort exhibited a significantly higher incidence of TTN than did the 38-week cohort. Conclusion: ECSs at 37 weeks of GA exhibited an increased risk of TTN than ECSs at ≥ 38 weeks of GA. Strategic neonatal care and adequate preparation can mitigate this risk without affecting the length of hospital stay.
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Alström syndrome is a form of inherited obesity caused by a single gene abnormality and is inherited as an autosomal recessive trait. It is characterised by a variety of clinical manifestations, including progressive visual and hearing impairment, type 2 diabetes mellitus, dilated cardiomyopathy, and hepatic and renal dysfunction, in addition to obesity. Recent insights underline the pivotal involvement of the disease-associated gene (ALMS1) in cilia formation and function, leading to the classification of its clinical manifestations as a ciliopathy. This review delineates the diverse clinical indicators defining the syndrome and elucidates its pathological underpinnings.
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Achondroplasia (ACH) is a rare, autosomal dominant skeletal dysplasia characterized by short stature, characteristic facial configuration, and trident hands. Before vosoritide approval in Japan, patients with ACH could start growth hormone (GH) treatment at age 3 years. However, ACH and its treatment in young Japanese children have not been studied. This retrospective, longitudinal, medical records-based cohort study (before vosoritide approval) summarized symptoms, complications, monitoring, surgery/interventions, and height with/without GH in Japanese patients with ACH <5 years. Complications were observed in 89.2% of all 37 patients; 75.7% required surgery or intervention. All patients were monitored by magnetic resonance imaging; 73.0% had foramen magnum stenosis, while 54.1% had Achondroplasia Foramen Magnum Score 3 or 4. Of 28 GH-treated patients, 22 initiating at age 3 years were generally taller after 12 months versus 9 non-GH-treated patients. Mean annual growth velocity significantly increased from age 2 to 3 versus 3 to 4 years in GH-treated patients (4.37 vs. 7.23 cm/year; p = 0.0014), but not in non-GH-treated patients (4.94 vs. 4.20 cm/year). The mean height at age 4 years with/without GH was 83.6/79.8 cm. These results improve our understanding of young patients with ACH in Japan and confirm that early diagnosis of ACH and monitoring of complications help facilitate appropriate interventions.
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Acondroplasia , Humanos , Acondroplasia/tratamiento farmacológico , Acondroplasia/genética , Acondroplasia/patología , Masculino , Femenino , Estudios Retrospectivos , Preescolar , Japón/epidemiología , Lactante , Hormona de Crecimiento Humana/uso terapéutico , Resultado del Tratamiento , Niño , Estatura/efectos de los fármacos , Manejo de la Enfermedad , Registros Médicos , Imagen por Resonancia Magnética , Pueblos del Este de AsiaRESUMEN
Burosumab, a fully human monoclonal antibody against fibroblast growth factor 23, is mainly administered to patients with severe X-linked hypophosphatemia (XLH). However, there have been few reports on its use in relatively mild cases. In this report, we administered burosumab to two siblings with XLH who had been effectively treated with oral phosphate and active vitamin D. Both patients showed further improvement in radiographic and laboratory findings with burosumab compared with conventional treatment. Upon switching treatment, popliteal pain was reported in case 1 until her phosphorus levels normalized. This emphasizes the importance of monitoring not only rickets and calcium/phosphate metabolism but all symptoms of XLH after initiating burosumab. Notably, in cases 1 and 2, burosumab sustained catch-up growth, especially in case 1, who had not yet reached puberty. Further clinical studies are needed to determine whether burosumab improves growth and proportional abnormalities in patients with mild XLH.
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BACKGROUND: In systemic inflammatory conditions, inflammatory cytokines can cause low thyroid hormone levels. There are no reports discussing the relation between thyroid hormone levels and response to treatment for Kawasaki disease. METHODS: We investigated 67 patients who underwent treatment in the acute phase of Kawasaki disease. We divided patients into two groups based on their response to initial intravenous immunoglobulin (IVIG) treatment: the responder group (n = 40), and the non-responder group (n = 27). The serum levels of the thyroid hormones free triiodothyronine (FT3), free thyroxine (FT4), and thyroid-stimulating hormone (TSH) were compared before and after treatment in all patients, and between responder and non-responder groups. RESULTS: The FT3, FT4, and TSH levels were low before the initial treatment and increased significantly after treatment (p < 0.05). The FT3, FT4, and TSH levels before treatment were significantly lower in the non-responder group than in the responder group (p < 0.05). Logistic regression analysis suggested that the addition of pre-treatment FT4 values to Gunma score was useful in predicting treatment failure. CONCLUSIONS: Thyroid hormone and TSH levels were lower in the non-responder group than in the responder group in the initial IVIG treatment for Kawasaki disease. This study suggests that Kawasaki disease in the acute phase is associated with low thyroid hormone levels and TSH. It is possible that these hormone levels predict response to the initial IVIG.
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Síndrome Mucocutáneo Linfonodular , Tiroxina , Humanos , Tiroxina/uso terapéutico , Síndrome Mucocutáneo Linfonodular/complicaciones , Síndrome Mucocutáneo Linfonodular/diagnóstico , Síndrome Mucocutáneo Linfonodular/tratamiento farmacológico , Inmunoglobulinas Intravenosas/uso terapéutico , Hormonas Tiroideas , TirotropinaRESUMEN
Tolvaptan, a vasopressin receptor antagonist, has been shown to be effective in the treatment of renal cysts in ADPKD. However, tolvaptan is not indicated for pediatric patients, and reports of its use are rare, making its efficacy and adverse reactions unclear. Herein, we present the case of an 11-year-old girl who had vitiligo from birth. She was diagnosed with West syndrome at 6 months of age and tuberous sclerosis at 2 years of age. At the age of 6 years, an abdominal magnetic resonance imaging (MRI) revealed multiple bilateral renal cysts, and she was diagnosed with ADPKD. Abdominal MRI scans performed at 10 years and 11 years showed rapid renal cyst enlargement, and the renal prognosis was judged to be poor. The patient was treated with tolvaptan to delay cyst exacerbation. There were no apparent adverse events after the initiation of treatment, and the MRI performed 12 months after treatment initiation showed that renal cyst enlargement was suppressed. The results suggest that tolvaptan may be effective in pediatric patients with severe ADPKD who have rapidly enlarging renal cysts, although evaluation of renal cyst enlargement and side effects should be continued.
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Background: Japan is reviewing how physicians operate and plans to implement a work-style reform for physicians in 2024. This study examined how outsourcing housework cleaning tasks changed the daily lives of university hospital physicians. Methods: A total of 18 physicians participated in the study, outsourcing cleaning tasks either once or thrice. Results: Fourteen out of 18 respondents reported a decrease in the burden of household chores. Additionally, 10 respondents reported having more time for family contact, and nine respondents reported having more time for their own hobbies and diversions. Meanwhile, only five respondents reported that they had more time to work. Conclusion: Outsourcing housework cleaning tasks and using the newly created time for family and self may improve work performance.
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OBJECTIVE: Coronavirus disease 2019 (COVID-19) has significantly impacted medical services worldwide. During the Omicron variant-predominant era, febrile seizure (FS) in patients with COVID-19 increased compared to that in the pre-Omicron variant era. Therefore, this study aimed to demonstrate the clinical characteristics of FS in patients with COVID-19. METHODS: We surveyed patients aged < 16 years who presented with FS to the emergency room of Tottori University Hospital. The patients were divided into two groups: FS patients with COVID-19 (FS with COVID-19 group) and FS patients without COVID-19 (FS without COVID-19 group) as per the results of the respiratory multiplex array test. Patients with positive results for both SARS-CoV-2 and other microorganisms were excluded. We obtained data on the patients' clinical backgrounds, symptoms, seizure duration, type of FS (simple or complex), diagnostic examinations, laboratory test results, and treatment. We compared the data between the FS with and without COVID-19 groups. RESULT: A total of 128 patients with FS met the inclusion criteria. Of these, 18 patients and 110 patients were included in the FS with COVID-19 group and without COVID-19 group, respectively. The late FS onset (>60 months) were significantly more common in the FS with COVID-19 group than that in the FS without COVID-19 group. Moreover, patients in the FS with COVID-19 group had significantly longer seizure durations than those in the FS without COVID-19 group. A diazepam (DZP) suppository was administered to 72% of FS patients with COVID-19 after the first seizure during a febrile episode. CONCLUSION: FS patients with COVID-19 had different distributions of age at onset and seizure duration than those without COVID-19. The use of DZP suppositories was more frequent in FS patients with COVID-19 compared to those without COVID-19.
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COVID-19 , Convulsiones Febriles , Niño , Humanos , Convulsiones Febriles/diagnóstico , COVID-19/complicaciones , SARS-CoV-2 , Diazepam/uso terapéuticoRESUMEN
Paired box protein 2 (PAX2) gene variant causes renal coloboma syndrome (MIM#120330). Further, they are associated with focal segmental glomerulosclerosis and characterized by basement membrane changes similar to Alport syndrome.Herein, we report an 8-year-old boy who presented with proteinuria and decreased renal function. His paternal uncle has focal segmental glomerulosclerosis and renal failure, and his paternal grandmother has renal failure and is receiving peritoneal dialysis. Further, his father has stage 2 chronic kidney disease. At 3 years of age, his serum creatinine-estimated glomerular filtration rate was 40-50 mL/min/1.73 m2. At 8 years of age, his renal function further decreased and he had proteinuria (urinary protein/Cr 3.39 g/g Cr). Renal histopathology showed oligonephronia and focal segmental glomerulosclerosis. A partial basket-weave pattern, similar to Alport syndrome, was also observed on a transmission electron microscope, and low-vacuum scanning electron microscopy revealed coarse meshwork changes in the glomerular basement membrane. Genetic analysis revealed a PAX2 heterozygous variant (NM_003987.4:c.959C > G), a nonsense variant in which the serine at position 320 changes to a stop codon, in our patient and his father. PAX2 is a transcription factor that is important for the podocyte variant. However, podocytes with PAX2 gene variants may cause abnormal basement membrane production and repair, thereby resulting in Alport-like changes.
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X-linked hypophosphatemia (XLH) is a rare, inherited, multisystem disorder characterized by hypophosphatemia that occurs secondary to renal phosphate wasting. Mutations in PHEX gene (located at Xp22.1) in XLH alter bone mineral metabolism, resulting in diverse skeletal, dental, and other extraskeletal abnormalities that become evident in early childhood and persist into adolescence and adult life. XLH impacts physical function, mobility, and quality of life, and is associated with substantial socioeconomic burden and health care resource utilization. As the burden of illness varies with age, an appropriate transition of care from childhood and adolescence to adulthood is necessary to meet growth-related changes and minimize long-term sequelae of the condition. Previous XLH guidelines that encompassed transition of care have focused on Western experience. Regional differences in resource availability warrant tailoring of recommendations to the Asia-Pacific (APAC) context. Hence, a core expert panel of 15 pediatric and adult endocrinologists from nine countries/regions across APAC convened to formulate evidence-based recommendations for optimizing XLH care. A comprehensive literature search on PubMed using MeSH and free-text terms relevant to predetermined clinical questions on diagnosis, multidisciplinary management, and transition of care of XLH revealed 2171 abstracts. The abstracts were reviewed independently by two authors to shortlist a final of 164 articles. A total of 92 full-text articles were finally selected for data extraction and drafting the consensus statements. Sixteen guiding statements were developed based on review of evidence and real-world clinical experience. The GRADE criteria were used to appraise the quality of evidence supporting the statements. Subsequently, a Delphi technique was utilized to rate the agreement on statements; 38 XLH experts (15 core, 20 additional, 3 international) from 15 countries/regions (12 APAC, 3 EU) participated in the Delphi voting to further refine the statements. Statements 1-3 cover the screening and diagnosis of pediatric and adult XLH; we have defined the clinical, imaging, biochemical, and genetic criteria and raised red flags for the presumptive and confirmatory diagnosis of XLH. Statements 4-12 tackle elements of multidisciplinary management in XLH such as therapeutic goals and options, composition of the multidisciplinary team, follow-up assessments, required monitoring schedules, and the role of telemedicine. Treatment with active vitamin D, oral phosphate, and burosumab is discussed in terms of applicability to APAC settings. We also expound on multidisciplinary care for different age groups (children, adolescents, adults) and pregnant or lactating women. Statements 13-15 address facets of the transition from pediatric to adult care: targets and timelines, roles and responsibilities of stakeholders, and process flow. We explain the use of validated questionnaires, desirable characteristics of a transition care clinic, and important components of a transfer letter. Lastly, strategies to improve XLH education to the medical community are also elaborated in statement 16. Overall, optimized care for XLH patients requires prompt diagnosis, timely multidisciplinary care, and a seamless transfer of care through the coordinated effort of pediatric and adult health care providers, nurse practitioners, parents or caregivers, and patients. To achieve this end, we provide specific guidance for clinical practice in APAC settings. © 2023 The Authors. JBMR Plus published by Wiley Periodicals LLC on behalf of American Society for Bone and Mineral Research.
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Although the heart is one of the most important organs for animal survival, its regenerative capacity varies among animal species. Notably, adult mammals cannot regenerate their hearts after damage such as acute myocardial infarction. In contrast, some vertebrate animals can regenerate the heart throughout their lives. Cross-species comparative studies are important to understand the full picture of cardiac regeneration in vertebrates. Among the animal species able to regenerate the heart, some urodele amphibians, such as newts, possess a remarkable capacity for this process. Standardized methods of inducing cardiac regeneration in the newt are needed as a platform for studies comparing newts and other animal models. The procedures presented here describe amputation and cryo-injury techniques for the induction of cardiac regeneration in Pleurodeles waltl, an emerging model newt species. Both procedures consist of simplified steps that require no special equipment. We additionally show some examples of the regenerative process obtained using these procedures. This protocol has been developed for P. waltl. However, these methods are also expected to be applicable to other newt and salamander species, facilitating comparative research with other model animals.
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Pleurodeles , Salamandridae , Animales , Vertebrados , MamíferosRESUMEN
CONTEXT: In an open-label, randomized, controlled, phase 3 trial in 61 children aged 1 to 12 years with X-linked hypophosphatemia (XLH), burosumab improved rickets vs continuing conventional therapy with active vitamin D and phosphate. OBJECTIVE: We conducted an analysis to determine whether skeletal responses differed when switching to burosumab vs continuing higher or lower doses of conventional therapy. METHODS: Conventional therapy dose groups were defined as higher-dose phosphate [greater than 40 mg/kg] (HPi), lower-dose phosphate [40 mg/kg or less] (LPi), higher-dose alfacalcidol [greater than 60 ng/kg] or calcitriol [greater than 30 ng/kg] (HD), and lower-dose alfacalcidol [60 ng/kg or less] or calcitriol [30 ng/kg or less] (LD). RESULTS: At week 64, the Radiographic Global Impression of Change (RGI-C) for rickets was higher (better) in children randomly assigned to burosumab vs conventional therapy for all prebaseline dose groups: HPi (+1.72 vs +0.67), LPi (+2.14 vs +1.08), HD (+1.90 vs +0.94), LD (+2.11 vs +1.06). At week 64, the RGI-C for rickets was also higher in children randomly assigned to burosumab (+2.06) vs conventional therapy for all on-study dose groups: HPi (+1.03), LPi (+1.05), HD (+1.45), LD (+0.72). Serum alkaline phosphatase (ALP) also decreased in the burosumab-treated patients more than in the conventional therapy group, regardless of on-study phosphate and active vitamin D doses. CONCLUSION: Prior phosphate or active vitamin D doses did not influence treatment response after switching to burosumab among children with XLH and active radiographic rickets. Switching from conventional therapy to burosumab improved rickets and serum ALP more than continuing either higher or lower doses of phosphate or active vitamin D.
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Raquitismo Hipofosfatémico Familiar , Hipofosfatemia , Niño , Humanos , Fosfatos , Anticuerpos Monoclonales/uso terapéutico , Vitamina D/uso terapéutico , Calcitriol/uso terapéutico , Vitaminas/uso terapéutico , Factores de Crecimiento de FibroblastosRESUMEN
Background: Achieving an optimal work-life balance (WLB) is an important social issue not only for workers in general but also for doctors due to the shortage of women doctors. The present study aims to survey doctors' WLB and their use of household chore support services (HCSS). Methods: A questionnaire survey was conducted with doctors working in Tottori Prefecture and a total of 289 responses (212 men, 77 women) were obtained and analyzed. To examine the relationship between gender and satisfaction with working patterns or with life for WLB, as well as the use of HCSS, a chi-squared test was conducted. Furthermore, a chi-squared test was conducted including age, marital status, whether or not they have preschool-aged children, and type of institution the participant worked. Results: A significant difference was found in type of institution the participant worked regarding satisfaction with work patterns and life. The proportion of those who have used HCSS was 12.5% of the total sample and was significantly higher for women than men (men: 8.5%; women: 23.4%). Regarding the reason to use HCSS, "to reduce the burden of household chores and childcare" and "to secure time for work" were most common with the same proportions, and amongst women, "to secure time for work" was the highest. Conclusion: Doctors working at a university hospital could have different work patterns and satisfaction with life compared to those working at other types of medical institutions. Additionally, the demand for HCSS was particularly high among women doctors, which suggests that HCSS may be used to reduce the burden of household chores and childcare, as well as to create time for work.
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Background: Extended-spectrum ß-lactamase (ESBL)-producing Escherichia coli has been increasingly recognized as the cause of upper urinary tract infection (UTI) in children. We have been using flomoxef at our department since 2017 as the first-line empiric therapy for children diagnosed with UTIs, and we avoid using carbapenems, which are considered the first-line treatment for ESBL-producing E. coli. However, reports on the use of flomoxef for UTIs are limited, especially for pediatric patients. The presence of vesicoureteral reflux at the onset of pyelonephritis is a concern. Severe vesicoureteral reflux can lead to repeated UTI and future deterioration of renal function, but the indication for voiding urethrography, which closely examines the presence of vesicoureteral reflux complications, is controversial. Methods: We retrospectively reviewed the laboratory findings, treatment, and clinical course of 96 pyelonephritis cases experienced at our department over a 7-year period from April 2014 to March 2021. Results: ESBL-producing E. coli were identified as the cause of pyelonephritis in 51% of cases, and this value was significantly higher (88%) in 2017. No significant differences were found in the febrile period or recurrence rate between the flomoxef-initiated group and other antibiotics groups. We also examined clinical indicators to predict vesicoureteral reflux and found no significant differences in ultrasonographic findings of hydronephrosis. Conclusion: In the present series, 51% of all pyelonephritis cases were found to be caused by ESBL-producing E. coli, with a significant increase in recent years. Flomoxef may be a useful alternative to carbapenem for ESBL-producing E. coli and the initial antibiotic of choice for upper UTIs in children. The indication for voiding cystourethrography should be carefully determined.
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INTRODUCTION: X-linked hypophosphataemia (XLH) is a rare, genetic renal phosphate-wasting disease, resulting from excess fibroblast growth factor 23 (FGF23) activity, which has a progressive and profound impact on patients throughout life. The monoclonal anti-FGF23 antibody, burosumab, is a subcutaneous injection indicated for the treatment of XLH in children and adults. Originally, burosumab was approved to be administered by a healthcare professional (HCP), but the option of self-administration would enable patient independence and easier access to treatment. Two open-label, single-arm clinical trials, conducted in Japan and Korea, have assessed the safety and efficacy of self-administration of burosumab in both children and adults with XLH. METHODS: In KRN23-003 (n = 15 children aged 1-12 years) and KRN23-004 (n = 5 children aged 3-13 years, n = 4 adults aged 21-65 years), children initially received 0.8 mg/kg of burosumab every 2 weeks and adults initially received 1.0 mg/kg of burosumab every 4 weeks. Self-administration was permitted from Week 4, and patients or carers were provided with training to inject correctly. RESULTS: In both trials, burosumab had an acceptable safety profile with mainly mild-to-moderate adverse events. Following self-administration, no patients reported serious treatment-emergent adverse events ≥ grade 3, injection-site reactions or hypersensitivity reactions related to burosumab. Serum phosphate and active vitamin D levels increased from baseline in children and adults. CONCLUSIONS: These results indicated that the efficacy and safety of burosumab when administered either by a carer or patient are similar to that when administered by an HCP and show that self-administration is a viable option for patients with XLH. TRIAL REGISTRATION NUMBERS: NCT03233126 and NCT04308096.
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Anticuerpos Monoclonales , Raquitismo Hipofosfatémico Familiar , Humanos , Adulto , Niño , Anticuerpos Monoclonales/efectos adversos , Raquitismo Hipofosfatémico Familiar/tratamiento farmacológico , Anticuerpos Monoclonales Humanizados/uso terapéutico , Fosfatos/uso terapéuticoRESUMEN
Background: Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infections were not prevalent in Yonago and its vicinity during autumn 2020, and the relative frequencies of pathogen-induced respiratory infections during this period are unclear. Methods: We collected 109 nasopharyngeal swabs from 93 pediatric patients who visited Tottori University Hospital between October 1, 2020, and March 31, 2021. These samples were comprehensively tested for 18 pathogens with the FilmArray® respiratory panel test (v2.1) using nested real-time polymerase chain reaction, and the frequency of pathogens detected per month was calculated. In addition, we compared the duration of fever and the blood test results of patients infected with each pathogen or multiple pathogens. Results: Of the 109 samples, 42 were obtained from female patients and 67 from male patients (median age, 3 years; range, 0-15 years). Overall, 62 patients (56.9%) had a fever ≥ 38 °C at the time of examination, and the median duration of fever ≥ 38 °C was 2 days (1-12). During the study period, the highest number of samples (22) were collected in November 2020. Among samples that tested positive, the most common pathogens were rhino/enteroviruses (52 samples; 76.5%), followed by adenoviruses (7 samples; 10.3%), coronavirus NL63 (6 samples; 8.8%), coronavirus OC43, parainfluenza virus type 1, and parainfluenza virus type 2 (1 sample each; 1.5% each). The duration of fever was significantly longer in adenovirus-infected patients than in patients infected with other viruses (P < 0.05). Hemoglobin and sodium levels were also significantly lower among the adenovirus-infected patients. However, these variations were mostly within the normal range. No clinically meaningful differences were found between rhino/enterovirus-infected and non-rhino/enterovirus-infected cases, between coronavirus NL63-infected and non-coronavirus NL63-infected cases, and between cases with multiple- and single-pathogen infections. Conclusion: Rhino/enteroviruses were the most common viruses causing respiratory tract infections in areas without endemic SARS-CoV-2.