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There are no agreed upon terminology to define "refractory" pediatric Immune Thrombocytopenia (ITP). Guidelines are therefore limited to arbitrary and outdated definitions. The Pediatric ITP Consortium of North America held a meeting in 2023 to define this entity. With 100% agreement, the faculty established that pediatric ITP that is refractory to emergent therapy could be defined as no platelet response after treatment with all eligible emergent pharmacotherapies. With 100% agreement, the working group established that pediatric patients with ITP that continue to demonstrate high disease burden and/or no platelet response despite treatment with multiple classes of disease modifying therapies represent a challenging subset of ITP. These patients are at higher risk of ongoing disease burden and merit additional investigation as well as consideration for clinical trials or novel therapies. Future efforts to define disease burden and disease response will be completed in collaboration with the ITP International Working Group.
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Globally, children have been adversely affected by the wide variety of impacts of SARS-CoV-2 (Coronavirus | COVID-19). Vulnerable children who depend on the support of education and health and social care systems were left unprotected as these systems were weakened by the pandemic. COVID-19 has exposed the already fragile situations in which many children and young people live and that thousands of children and young people would need ongoing support but remained invisible to statutory authorities. COVID-19 has also been a stark reminder of the vulnerability of individuals and societies and it has exposed deep divisions, inequalities, and injustices between different countries and groups of people. M'Lop Tapang is a local nonprofit organization registered with the Royal Government of Cambodia. This report discusses the efforts of M'Lop Tapang following the declaration of the COVID-19 global pandemic in early 2020, to address the local community needs; to ensure the voices of children remained heard; and to promote children's rights remaining a priority throughout the remainder of the pandemic.What is already known on this topicVulnerable children are dependent on remaining visible to education and health and social care systems to ensure they are safeguarded and protected.Poor safety, financial instability, unemployment, challenges to children's rights and displacement all have the potential to increase vulnerabilities and intensify inequalities.The COVID-19 global pandemic has widened the global lens for the nursing profession and to effect change, children's nurses need to think globally and act locally.What this study addsThis study provides insights into the challenges faced by children and families in Sihanoukville, Cambodia, during the early part of the SARS-CoV-2 (COVID19) pandemic in 2020-2021.The economic impact of COVID-19 on children and families living in Sihanoukville, Cambodia, between 2020 and 2021 is reviewed.The learning from the pragmatic and rapid interventions of M'Lop Tapang, Sihanoukville during the early phase of the SARS-CoV-2 (COVID-19) pandemic, are of use in other countries around the world in future epidemic or pandemic situations.
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COVID-19 , Humanos , COVID-19/epidemiología , COVID-19/prevención & control , Cambodia , Niño , Adolescente , SARS-CoV-2 , PandemiasRESUMEN
ABSTRACT: Despite the global unrelated donor (URD) registry size, the degree to which URD availability is a transplant barrier is not established. We evaluated the availability of 3,843 URDs requested for 455 diverse adult patients (predominantly with acute leukemia). URDs for non-Europeans were more likely to be domestic and had markedly lower Donor Readiness scores. Of URDs requested for confirmatory HLA-typing (CT) alone (ie, without simultaneous workup), 1,894 of 3,529 (54%) were available. Availability of domestic URDs was 45%. Donor Readiness score was highly predictive of CT availability. More non-European patients (n = 120) than Europeans (n = 335) had >10 URDs requested and <5 available. Of workup requests (after CT or CT-workup), <70% (604/889 [68%]) were available. More non-Europeans had <2 URDs available. URD availability for CT was markedly worse for non-Europeans, with availabilities for African, non-Black Hispanic, and Asian patients being 150/458 (33%), 120/258 (47%), and 119/270 (44%), respectively, with further decrements in URD workup availability. Our data suggest the functional size of the URD pool is much smaller than appreciated, mandating major operational changes for transplant centers and donor registries. Likelihood of donor availability should have a high priority in donor selection. Considering patient ancestry and URD Donor Readiness scores, centers should pursue, and registries permit, simultaneous pursuit of many URDs and abandon futile searches. Patients should be informed about their likelihood of donor availability and alternative options. Finally, although registries should address high URD attrition and speed procurement, use of all HLA-disparate graft types is needed to facilitate timely transplant for all.
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Trasplante de Células Madre Hematopoyéticas , Donante no Emparentado , Humanos , Donante no Emparentado/provisión & distribución , Masculino , Trasplante Homólogo , Femenino , Adulto , Etnicidad , Sistema de Registros , Persona de Mediana Edad , Grupos Raciales , VoluntariosRESUMEN
The presentation of immune thrombocytopenia is dependent on the degree of thrombocytopenia, with no to mild bleeding symptoms, primarily mucocutaneous bleeding. Severe bleeding in other organ systems is a rare complication. Spontaneous hemarthrosis is rare in patients without hemophilia. We report a child presenting with oral and cutaneous petechial lesions and left knee hemarthrosis without trauma. Laboratory findings showed severe thrombocytopenia consistent with immune thrombocytopenia. Serologic tests were consistent with Lyme disease. Hemarthrosis was presumed secondary to Lyme disease monoarticular joint inflammation with bleeding exacerbated by severe thrombocytopenia. Hemarthrosis resolved and platelet counts normalized following immunoglobulin infusion, steroid course, and antibiotics.
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Hemofilia A , Enfermedad de Lyme , Púrpura Trombocitopénica Idiopática , Humanos , Niño , Hemartrosis/complicaciones , Hemartrosis/diagnóstico , Púrpura Trombocitopénica Idiopática/complicaciones , Enfermedad de Lyme/complicaciones , Enfermedad de Lyme/diagnóstico , Enfermedad de Lyme/tratamiento farmacológico , Hemofilia A/complicaciones , Antibacterianos/uso terapéuticoRESUMEN
Despite the prevalence of dysautonomia in people with Long COVID, it is currently unknown whether Long COVID dysautonomia is routinely accompanied by structural or functional cardiac alterations. In this retrospective observational study, the presence of echocardiographic abnormalities was assessed. Left ventricular (LV) chamber sizes were correlated to diagnostic categories and symptoms via standardized patient-reported outcome (PRO) questionnaires. A total of 203 individuals with Long COVID without pre-existing cardiac disease and with available echocardiograms were included (mean age, 45 years; 67% female). Overall, symptoms and PRO scores for fatigue, breathlessness, quality of life, disability, anxiety and depression were not different between those classified with post-COVID dysautonomia (PCD, 22%) and those unclassified (78%). An LV internal diameter at an end-diastole z score < -2 was observed in 33 (16.5%) individuals, and stroke volume (SV) was lower in the PCD vs. unclassified subgroup (51.6 vs. 59.2 mL, 95% C.I. 47.1-56.1 vs. 56.2-62.3). LV end-diastolic volume (mean diff. (95% CI) -13 [-1--26] mL, p = 0.04) and SV (-10 [-1--20] mL, p = 0.03) were smaller in those individuals reporting a reduction in physical activity post-COVID-19 infection, and smaller LVMI was weakly correlated with worse fatigue (r = 0.23, p = 0.02). The majority of individuals with Long COVID report shared symptoms and did not demonstrate cardiac dysfunction on echocardiography.
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Patients who are hospitalized due to COVID-19 are predisposed to requiring acute inpatient rehabilitation. Multiple factors have posed challenges to inpatient rehabilitation during the COVID-19 pandemic, such as staff shortages, restrictions with therapy, and barriers to discharge. Despite these challenges, data have shown that inpatient rehabilitation plays a key role in functional gains for this patient population. There remains a need for more data on the current challenges that are faced in the inpatient rehabilitation setting, as well as better understanding of long-term functional outcomes following COVID-19.
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COVID-19 , Humanos , Pacientes Internos , PandemiasRESUMEN
OBJECTIVES: Iron deficiency (ID) with and without anemia is prevalent in children and adults diagnosed with inflammatory bowel disease (IBD), but often goes unrecognized. We hypothesized, quality improvement (QI) methodology could increase the screening for and treatment of ID in children newly diagnosed with IBD. METHODS: We developed and implemented an easy-to-follow algorithm to facilitate screening for and treatment of ID for patients diagnosed with IBD. Through a series of Plan-Do-Study-Act cycles, the approach was modified to increase screening and treatment of ID. Data between January 2019 and July 2021 were assessed using statistical process control. RESULTS: Among patients newly diagnosed with IBD, 298 patients were included (67% Crohn disease, 29% ulcerative colitis, 4% indeterminate colitis, and 56% males). Rates of ID screening increased significantly from a baseline of 20% to >90%. Of the 232 patients screened for ID during the improvement period, 205 (88%) met criteria for either iron deficiency anemia (IDA) or ID at diagnosis, specifically, 151 (65%) met criteria for IDA and 54 (23%) met criteria for ID. CONCLUSIONS: Use of QI methodology to standardize screening assessments for ID among children newly diagnosed with IBD improved screening rates from a baseline of 20% to >90%, with 88% of patients found to have IDA or ID.
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Anemia Ferropénica , Anemia , Colitis Ulcerosa , Enfermedad de Crohn , Enfermedades Inflamatorias del Intestino , Deficiencias de Hierro , Masculino , Adulto , Niño , Humanos , Adolescente , Femenino , Enfermedades Inflamatorias del Intestino/complicaciones , Colitis Ulcerosa/complicaciones , Colitis Ulcerosa/diagnóstico , Enfermedad de Crohn/complicaciones , Enfermedad de Crohn/diagnóstico , Anemia Ferropénica/diagnóstico , Anemia Ferropénica/etiología , Anemia Ferropénica/terapiaRESUMEN
OBJECTIVE: This study evaluated sex differences in performance on the Sport Concussion Assessment Tool-5 (SCAT5) Standardized Assessment of Concussion (SAC) and in baseline SCAT5 symptom reporting. It established clinically relevant cut points for low performance on the SAC based on both reliable chance indices (RCIs) and normative performance. This study also evaluated the diagnostic utility of the sex-adjusted SCAT5 SAC for identification of suspected concussion in collegiate athletes. METHOD: In total, 671 uninjured collegiate athletes were administered the SCAT5 and 264 of these athletes also completed SCAT5 testing ~1 year later. Fifty-four athletes were administered the SCAT5 after being removed from play due to suspected concussion. Sex differences in cognitive performance and symptom reporting at baseline were evaluated and sex-specific clinically relevant cut points were provided. Chi square and logistic regression models were used to evaluate if SAC performance was a significant predictor of concussion status. RESULTS: Female athletes outperformed male athletes on the SCAT 5 SAC and showed minimally higher symptom endorsement. Use of sex-corrected normative data improved performance of the SAC in identification of suspected concussion when a low score cut point was used. Logistic regression models showed that sex-corrected SAC change from baseline (RCI) improved the predictive value of the model after first accounting for other elements of the SCAT5. CONCLUSIONS: Present results support the use of sex-specific normative data for the SCAT5 SAC, particularly if using low performance without comparison to a baseline; however, reliable change from a pre-injury baseline may have somewhat higher diagnostic utility.
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Traumatismos en Atletas , Conmoción Encefálica , Deportes , Femenino , Masculino , Humanos , Traumatismos en Atletas/diagnóstico , Caracteres Sexuales , Pruebas Neuropsicológicas , Conmoción Encefálica/diagnóstico , Conmoción Encefálica/psicología , AtletasRESUMEN
Examining behavior within the context of applied behavior analysis (ABA) from the perspective of acceptance and commitment training (ACTr), while understanding the role of relational framing and the transformation of stimulus function, can better equip behavior analysts to effectively address complex behavior that may be influenced by covert verbal behavior. This approach may lead to a more comprehensive analysis of behavior and result in more effective interventions for socially significant change. The aim of this article is to provide behavior analysts with information and guidance on using relational frame theory (RFT) and ACTr in behavior-analytic services. The article first provides a rationale for considering thoughts as behavior and outlines important components of RFT. Subsequently, the benefits of employing ACTr in ABA are highlighted, including brief descriptions and examples of the use of ACTr within ABA practice. The article highlights the importance of expanding the consideration of maintaining variables when attempting to address maladaptive covert verbal behavior and ultimately aims to encourage a greater number of behavior analysts to adopt RFT and ACTr practices in applied settings.
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The female athlete triad represents the 3 interrelated components: of energy availability (EA), menstrual function and bone health. Each component exists on a spectrum ranging from optimal health to dysfunction. Screening for the triad during the annual wellness exam, the preparticipation physical evaluation (PPE) or when the athlete presents with any single component can help identify athletes at risk. A multidisciplinary team is helpful in managing the treatment of the Triad which relies on improving EA. Screening, early recognition and aggressive treatment is important, especially in adolescent athletes to optimize bone health.
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BACKGROUND: The majority of older people (> 65 years) in hospital have frailty and are at increased risk of readmission or death following discharge home. In the UK, following acute hospitalisation, around one third of older people with frailty are referred on for rehabilitation, termed 'intermediate care' services. Although this rehabilitation can reduce early readmission to hospital (< 30 days), recipients often do not feel ready to leave the service on discharge, suggesting possible incomplete recovery. Limited evidence suggests extended rehabilitation is of benefit in several conditions and there is preliminary evidence that progressive physical exercise can improve mobility and function for older people with frailty, and slow progression to disability. Our aim is to evaluate the effectiveness of the Home-based Older People's Exercise (HOPE) programme as extended rehabilitation for older people with frailty discharged home from hospital or intermediate care services after acute illness or injury. METHODS: A multi-centre individually randomised controlled trial, to evaluate the clinical and cost-effectiveness of the HOPE programme. This individualised, graded and progressive 24-week exercise programme is delivered by NHS physiotherapy teams to people aged 65 and older with frailty, identified using the Clinical Frailty Scale, following discharge from acute hospitalisation and linked intermediate care rehabilitation pathways. The primary outcome is physical health-related quality of life, measured using the physical component summary score of the modified Short Form 36- item health questionnaire (SF36) at 12 months. Secondary outcomes include self-reported physical and mental health, functional independence, death, hospitalisations, care home admissions. Plans include health economic analyses and an embedded process evaluation. DISCUSSION: This trial seeks to determine if extended rehabilitation, via the HOPE programme, can improve physical health-related quality of life for older people with frailty following acute hospitalisation. Results will improve awareness of the rehabilitation needs of older people with frailty, and provide evidence on the clinical and cost-effectiveness of the targeted exercise intervention. There is potential for considerable benefit for health and social care services through widespread implementation of trial findings if clinical and cost-effectiveness is demonstrated. TRIAL REGISTRATION: ISRCTN 13927531 . Registered on April 19, 2017.
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Fragilidad , Enfermedad Aguda , Anciano , Análisis Costo-Beneficio , Terapia por Ejercicio , Fragilidad/diagnóstico , Humanos , Estudios Multicéntricos como Asunto , Calidad de Vida , Ensayos Clínicos Controlados Aleatorios como AsuntoRESUMEN
The methodologic approach used in next-generation sequencing (NGS) affords a high depth of coverage in genomic analysis. Inherent in the nature of genomic testing, there exists potential for identifying genomic findings that are incidental or secondary to the indication for clinical testing, with the frequency dependent on the breadth of analysis and the tissue sample under study. The interpretation and management of clinically meaningful incidental genomic findings is a pressing issue particularly in the pediatric population. Our study describes a 16-mo-old male who presented with profound global delays, brain abnormality, progressive microcephaly, and growth deficiency, as well as metopic craniosynostosis. Clinical exome sequencing (ES) trio analysis revealed the presence of two variants in the proband. The first was a de novo variant in the PPP2R1A gene (c.773G > A, p.Arg258His), which is associated with autosomal dominant (AD) intellectual disability, accounting for the proband's clinical phenotype. The second was a recurrent hotspot variant in the CBL gene (c.1111T > C, p.Tyr371His), which was present at a variant allele fraction of 11%, consistent with somatic variation in the peripheral blood sample. Germline pathogenic variants in CBL are associated with AD Noonan syndrome-like disorder with or without juvenile myelomonocytic leukemia. Molecular analyses using a different tissue source, buccal epithelial cells, suggest that the CBL alteration may represent a clonal population of cells restricted to leukocytes. This report highlights the laboratory methodologic and interpretative processes and clinical considerations in the setting of acquired variation detected during clinical ES in a pediatric patient.
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Hallazgos Incidentales , Discapacidad Intelectual , Niño , Genómica , Secuenciación de Nucleótidos de Alto Rendimiento , Humanos , Masculino , FenotipoRESUMEN
INTRODUCTION: CKD, a common complication of type-2 diabetes (T2D), causes considerable disease burden. Patients with T2D and CKD are considered high-risk for complications; however, studies describing patients with T2D and incident CKD identified from real-world data using the diagnostic gold-standard criteria - estimated glomerular filtration rate and urine albumin-to-creatinine ratio (UACR) - are scarce. METHODS: In this population-based cohort study, we sought to estimate the rates of cardiovascular and renal outcomes among patients with T2D and CKD by comorbidity subgroups and CKD severity. Patients were sampled between 2008 and 2017 from de-identified US administrative claims enriched with laboratory data. Analyses were stratified by prevalent heart failure (HF), anemia, and resistant hypertension and the KDIGO categories at index. RESULTS: We identified 106,369 patients with T2D and incident CKD. The rate of all-cause hospitalization was 189 [95% CI: 187, 191] per 1,000 person-years with cardiovascular-related hospitalizations being more frequent than kidney-related outcomes. The rate of acute kidney failure was 77.3 [95% CI: 76.2, 78.5] per 1,000 person-years. Patients with HF experienced a 4-times higher rate for cardiovascular events compared to those without. Rates of hospitalization increased from 5- to 6-fold with increasing KDIGO severity. CONCLUSIONS: Multimorbidity and advance stages of CKD increase the risk of cardiovascular and renal complications among patients with T2D diabetes. Earlier CKD diagnosis as well as interventions and coordinated care addressing other comorbid conditions present at diagnosis may reduce the overall disease burden in this population.
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Enfermedades Cardiovasculares/epidemiología , Enfermedades Cardiovasculares/etiología , Diabetes Mellitus Tipo 2/complicaciones , Riñón/fisiopatología , Insuficiencia Renal Crónica/complicaciones , Anciano , Anciano de 80 o más Años , Albúminas/análisis , Anemia/epidemiología , Anemia/etiología , Estudios de Cohortes , Creatinina/sangre , Femenino , Tasa de Filtración Glomerular , Insuficiencia Cardíaca/epidemiología , Insuficiencia Cardíaca/etiología , Hospitalización/estadística & datos numéricos , Humanos , Hipertensión Renal/epidemiología , Hipertensión Renal/etiología , Masculino , Persona de Mediana Edad , Insuficiencia Renal Crónica/fisiopatología , Resultado del Tratamiento , Estados Unidos/epidemiologíaRESUMEN
OBJECTIVES: Agitation is common and problematic in care home residents with dementia. This study investigated the (cost)effectiveness of Dementia Care Mapping™ (DCM) for reducing agitation in this population. METHOD: Pragmatic, cluster randomised controlled trial with cost-effectiveness analysis in 50 care homes, follow-up at 6 and 16 months and stratified randomisation to intervention (n = 31) and control (n = 19). Residents with dementia were recruited at baseline (n = 726) and 16 months (n = 261). Clusters were not blinded to allocation. Three DCM cycles were scheduled, delivered by two trained staff per home. Cycle one was supported by an external DCM expert. Agitation (Cohen-Mansfield Agitation Inventory (CMAI)) at 16 months was the primary outcome. RESULTS: DCM was not superior to control on any outcomes (cross-sectional sample n = 675: 287 control, 388 intervention). The adjusted mean CMAI score difference was -2.11 points (95% CI -4.66 to 0.44, p = 0.104, adjusted ICC control = 0, intervention 0.001). Sensitivity analyses supported the primary analysis. Incremental cost per unit improvement in CMAI and QALYs (intervention vs control) on closed-cohort baseline recruited sample (n = 726, 418 intervention, 308 control) was £289 and £60,627 respectively. Loss to follow-up at 16 months in the original cohort was 312/726 (43·0%) mainly (87·2%) due to deaths. Intervention dose was low with only a quarter of homes completing more than one DCM cycle. CONCLUSION: No benefits of DCM were evidenced. Low intervention dose indicates standard care homes may be insufficiently resourced to implement DCM. Alternative models of implementation, or other approaches to reducing agitation should be considered.
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Demencia , Estudios de Cohortes , Análisis Costo-Beneficio , Estudios Transversales , Demencia/terapia , Humanos , Agitación Psicomotora/terapia , Calidad de VidaRESUMEN
Up to two-thirds of menstruating women experience abnormal uterine bleeding (AUB) when treated with oral anticoagulants. However, the true prevalence of AUB for specific agents remains uncertain, as many of these episodes, while interfering significantly with quality of life and overall health, are not captured by definitions of major bleeding (MB) or clinically relevant nonmajor bleeding (CRNMB) used in clinical trials. A 2017 systematic review determined that women taking rivaroxaban, but not edoxaban or apixaban, had a twofold higher risk of AUB than women taking warfarin. Since then, new data have become available from extension trials, cancer-associated venous thromboembolism trials, pediatric trials, and a few observational studies specifically examining AUB as an outcome. Reported rates of uterine CRNMB were low (around 1%) and similar for rivaroxaban and apixaban in all these studies, and no episodes of uterine bleeding meeting MB criteria were reported. Rates of AUB not meeting MB or CRNMB criteria were much higher, affecting up to 50% of women on rivaroxaban. Only 1 such study included women on apixaban, and no AUB was reported. In pediatric trials, 19% of girls experienced menorrhagia when treated with rivaroxaban. In conclusion, rates of uterine MB and CRNMB were low in all studies, but rates of other types of AUB not meeting these criteria ranged from 15.8% to 50%. We conclude that AUB is underreported due to the limitations of MB/CRNMB criteria despite its substantial impact on quality of life. We urge future investigators to include broader definitions of AUB to better capture the impact of this outcome in menstruating women treated with oral anticoagulants.