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BACKGROUND: Sexually transmitted infections (STIs) are common and disproportionately affect Black adolescents and young adults (AYAs). Less is known about STIs among Black AYAs with chronic conditions, such as sickle cell disease (AYAs-SCD). This study compared STI testing and diagnosis between AYAs-SCD and their peers, overall and among STI-related encounters. PROCEDURE: This retrospective, cross-sectional study used diagnosis and billing codes in the Pediatric Health Information System (PHIS) to identify inpatient and emergency department encounters from January 1, 2022 to May 31, 2023 among all AYAs 15-24 years and those with STI-related diagnoses (e.g., "cystitis"). STI testing and diagnosis rates were compared between AYAs-SCD, non-Black AYAs, and Black AYAs, controlling for age, sex, and encounter setting. RESULTS: We identified 3602 AYAs-SCD, 177,783 Black AYAs, and 534,495 non-Black AYAs. AYAs-SCD were less likely to be tested for STIs than non-Black AYAs (odds ratio [OR] = 0.26; adj. p < .001) and Black AYAs (OR = 0.53; adj. p < .001). When tested, AYAs-SCD were more likely to be diagnosed with an STI than non-Black AYAs (OR = 2.39; adj. p = .006) and as likely as Black AYAs (OR = 0.67; adj. p = .15). Among STI-related encounters, AYAs-SCD were less likely to be tested than non-Black AYAs (OR = 0.18; adj. p < .001) and Black AYAs (OR = 0.44; adj. p < .001). No significant differences in STI diagnoses were found in this subset between AYAs-SCD and non-Black AYAs (OR = 0.32; adj. p = .28) or Black AYAs (OR = 1.07; adj. p = .99). CONCLUSIONS: STI care gaps may disproportionately affect AYAs-SCD. STIs should be considered when evaluating symptomatic AYAs-SCD in acute settings. More research is needed to further contextualize STI care for AYAs-SCD.
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Influenza vaccination is critical for children with sickle cell disease (SCD) due to risks of severe influenza infections. Despite declining influenza vaccination among children since the COVID-19 pandemic, less is known about influenza vaccine coverage among youth with SCD during this same period. We compared influenza vaccine uptake among youth with SCD seen by a SCD provider in clinic during the 2019-2020 and 2022-2023 influenza seasons and described infection characteristics. Overall, 85% (n = 220) of children received their influenza vaccine during 2019-2020 compared to 75% (n = 245) in 2022-2023 (p = 0.059). Participants seen during both seasons were more likely to shift from vaccinated in 2019-2020 to unvaccinated in 2022-2023 than vice versa (McNemar's OR = 3.0; p = 0.008). Among 66 documented infections, 25.8% resulted in hospitalization. We found high influenza vaccine uptake but those seen during both seasons were more likely to become unvaccinated. More research is needed to understand influenza vaccine decision-making in this population.
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Current studies describing younger children with Hodgkin lymphoma are limited by geographical region, small sample sizes and variable age groups. Although published data is lacking, there appears to be a trend toward a higher male to female ratio and a higher proportion of mixed cellularity subtype when compared to older cohorts. We performed a retrospective multicenter study utilizing the Pediatric Health Information System® database to evaluate patients aged 0-39 years with Hodgkin lymphoma. We identified 3,034 unique patients who met inclusion criteria. Younger age groups had a larger proportion of males, Hispanic/Latino ethnicity, and mixed cellularity subtype. Treatment-related complications, including mucositis, pain, bacterial infections, and thrombosis, were documented more frequently in older cohorts. We also found significant age-related differences in medical management. This study is the largest study evaluating age-related differences in patients with Hodgkin lymphomaand the first study to evaluate for differences in complicationsand supportive care management.
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Enfermedad de Hodgkin , Humanos , Enfermedad de Hodgkin/epidemiología , Adolescente , Masculino , Femenino , Niño , Preescolar , Estudios Retrospectivos , Lactante , Adulto , Factores de Edad , Adulto Joven , Recién NacidoRESUMEN
Consistent with studies showing a high prevalence of the Duffy null phenotype among healthy Black Americans, this retrospective study found that Duffy null was present in >75% of a young and contemporary cohort of children with sickle cell disease (SCD) in the United States. Despite the potential for this phenotype to impact absolute neutrophil counts, hydroxyurea (HU) dosing, and outcomes, it was not associated with being prescribed a lower HU dose or having increased acute SCD visits early in the HU treatment course. Future studies are needed to confirm these findings in older children with SCD.
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Anemia de Células Falciformes , Antidrepanocíticos , Sistema del Grupo Sanguíneo Duffy , Hidroxiurea , Humanos , Hidroxiurea/uso terapéutico , Hidroxiurea/administración & dosificación , Anemia de Células Falciformes/tratamiento farmacológico , Anemia de Células Falciformes/epidemiología , Masculino , Femenino , Estudios Retrospectivos , Preescolar , Estados Unidos/epidemiología , Niño , Sistema del Grupo Sanguíneo Duffy/genética , Prevalencia , Antidrepanocíticos/uso terapéutico , Lactante , Receptores de Superficie Celular/genética , AdolescenteRESUMEN
Background: Patients with relapsed intracranial germinoma can achieve durable remission with standard chemotherapy regimens and/or reirradiation; however, innovative therapies are required for patients with relapsed and/or refractory intracranial nongerminomatous germ cell tumors (NGGCTs) due to their poor prognosis. Improved outcomes have been reported using reinduction chemotherapy to achieve minimal residual disease, followed by marrow-ablative chemotherapy (HDCx) with autologous hematopoietic progenitor cell rescue (AuHPCR). We conducted a phase II trial evaluating the response and toxicity of a 3-drug combination developed for recurrent intracranial germ cell tumors consisting of gemcitabine, paclitaxel, and oxaliplatin (GemPOx). Methods: A total of 9 patients with confirmed relapsed or refractory intracranial GCT were enrolled after signing informed consent, and received at least 2 cycles of GemPOx, of which all but 1 had relapsed or refractory NGGCTs. One patient with progressive disease was found to have pathologically confirmed malignant transformation to pure embryonal rhabdomyosarcoma (without GCT elements), hence was ineligible and not included in the analysis. Patients who experienced sufficient responses proceeded to receive HDCx with AuHPCR. Treatment response was determined based on radiographic tumor assessments and tumor markers. Results: A total of 7 patients achieved sufficient response and proceeded with HDCx and AuHPCR, and 5 subsequently received additional radiotherapy. A total of 2 patients developed progressive disease while receiving GemPOx. Myelosuppression and transaminitis were the most common treatment-related adverse events. With a mean follow-up of 44 months, 4 patients (3 NGGCTs, 1 germinoma) are alive without evidence of disease. Conclusions: GemPOx demonstrates efficacy in facilitating stem cell mobilization, thus facilitating the feasibility of both HDCx and radiotherapy.
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Background: Cancer is associated with increased venous thromboembolism in children. Risk factors for venous thromboembolism in this cohort include using central venous catheters, mass effect from underlying malignancy, chemotherapy, and surgery. Anticoagulation management in this cohort is challenging, given recurrent episodes of thrombocytopenia, the need for invasive procedures, and coagulopathy. A quality improvement (QI) initiative was developed to improve hematology consultation services and provide documentation of an individualized anticoagulation care plan for this high-risk cohort. Methods: Through the use of QI methods, interviews of stakeholders, expert consensus, and review of baseline data, a multidisciplinary team was organized, and key drivers relevant to improving access to hematology consultations and documentation of individualized anticoagulation care plans were identified. We used a Plan-Do-Study-Act model to improve hematology consultations and documentation of anticoagulation care plan (process measure). Outcome measures were bleeding and thrombosis recurrence/progression. Results: Seventeen patients with oncologic and venous thromboembolism diagnoses were included as baseline data. Slightly over half of these patients [53% (n = 9)] had a hematology consultation, and 7 (43.8%) had documentation of an anticoagulation care plan. After implementing QI methods, all 34 patients (100%) received hematology consultations and documentation of an anticoagulation care plan, and this measure was sustained for 1 year. Bleeding and thrombosis rates were similar in the baseline and post-QI cohorts. Conclusions: QI interventions proved effective in sustaining access to hematology consultations and providing anticoagulation care plans for patients with concomitant improved anticoagulation plan documentation for patients.
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INTRODUCTION: Surveillance following sacrococcygeal teratoma (SCT) resection varies. The purpose of this study was to describe the clinical characteristics and outcomes of patients undergoing SCT resection and examine current institutional practices to detect recurrence. METHODS: A single-institution retrospective review of children who underwent resection of an SCT from January 1, 2010 to December 31, 2020 was performed. Data were summarized and surveillance strategies compared between histopathologic subtypes using nonparametric methods. RESULTS: Thirty six patients (75.0% female) underwent SCT removal at a median age of 8 d. Histopathology revealed 27 mature teratomas (75.0%), eight immature teratomas (22.2%), and one malignant germ cell tumor (2.8%). Median postoperative follow-up was 3.17 y (interquartile range [IQR]: 2.31-4.38 y). Patients had a median of 2.32 clinic visits per year (IQR: 2.00-2.70), alpha-fetoprotein levels were obtained at a median of 2.01 times per year (IQR: 0-1.66), and surveillance imaging was performed at a median of 2.31 times per year (IQR: 0-2.84). Patients with immature teratomas had alpha-fetoprotein laboratories obtained more frequently than patients with mature teratomas (3.10 times/year versus 0.93 times/year, P = 0.001). There was no significant difference in the number of imaging studies obtained between groups. Two patients (5.6%) developed recurrence, which were identified on magnetic resonance imaging at 191 and 104 d postresection, respectively. CONCLUSIONS: Postoperative surveillance practices varied widely. Recurrence was noted in a single malignant case in the first year following resection. Multi-institutional studies are needed to determine the optimal surveillance strategy to detect recurrence of SCT.
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Neoplasias de Células Germinales y Embrionarias , Neoplasias Pélvicas , Teratoma , Niño , Humanos , Femenino , Masculino , alfa-Fetoproteínas , Región Sacrococcígea/patología , Región Sacrococcígea/cirugía , Teratoma/diagnóstico por imagen , Teratoma/cirugía , Neoplasias de Células Germinales y Embrionarias/patología , Neoplasias Pélvicas/patologíaRESUMEN
OBJECTIVE: To determine the impact of nodal basin ultrasound (US) surveillance versus completion lymph node dissection (CLND) in children and adolescents with sentinel lymph node (SLN) positive melanoma. BACKGROUND: Treatment for children and adolescents with melanoma are extrapolated from adult trials. However, there is increasing evidence that important clinical and biological differences exist between pediatric and adult melanoma. METHODS: Patients ≤18 years diagnosed with cutaneous melanoma between 2010 and 2020 from 14 pediatric hospitals were included. Data extracted included demographics, histopathology, nodal basin strategies, surveillance intervals, and survival information. RESULTS: Of 252 patients, 90.1% (n=227) underwent SLN biopsy (SLNB), 50.9% (n=115) had at least 1 positive node. A total of 67 patients underwent CLND with 97.0% (n=65/67) performed after a positive SLNB. In contrast, 46 total patients underwent US observation of nodal basins with 78.3% (n=36/46) of these occurring after positive SLNB. Younger patients were more likely to undergo US surveillance (median age 8.5 y) than CLND (median age 11.3 y; P =0.0103). Overall, 8.9% (n=21/235) experienced disease recurrence: 6 primary, 6 nodal, and 9 distant. There was no difference in recurrence (11.1% vs 18.8%; P =0.28) or death from disease (2.2% vs 9.7%; P =0.36) for those who underwent US versus CLND, respectively. CONCLUSIONS: Children and adolescents with cutaneous melanoma frequently have nodal metastases identified by SLN. Recurrence was more common among patients with thicker primary lesions and positive SLN. No significant differences in oncologic outcomes were observed with US surveillance and CLND following the identification of a positive SLN.
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Melanoma , Ganglio Linfático Centinela , Neoplasias Cutáneas , Adulto , Humanos , Adolescente , Niño , Melanoma/diagnóstico por imagen , Melanoma/cirugía , Melanoma/patología , Neoplasias Cutáneas/diagnóstico por imagen , Neoplasias Cutáneas/cirugía , Ganglio Linfático Centinela/patología , Recurrencia Local de Neoplasia/patología , Escisión del Ganglio Linfático , Biopsia del Ganglio Linfático Centinela , Estudios RetrospectivosRESUMEN
INTRODUCTION: Prompt, appropriate coagulation factor replacement according to injury and bleeding severity in persons with haemophilia is required to prevent acute and long-term complications. AIMS: Increase proportion of persons with haemophilia A (HA) and B (HB) treated appropriately for an acute injury and bleeding episode at a tertiary children's emergency department (ED) from 65% to 85% and sustain for one year. Secondary aim: increase time interval between patient ED encounters with out-of-range factor dosing. METHODS: Utilizing quality improvement methodology and plan-do-study-analyze cycles, ED encounters for individuals with HA/HB receiving coagulation factor replacement for injuries were audited for in-range coagulation factor dosing. Goal factor dose defined as 50% correction for minor bleeds and 100% correction for major bleeds. Optimal dosing range defined as 90%-120% of the calculated goal dose to account for vial size variability. Interventions targeted communication via the EMR problem list and optimization of physician education. RESULTS: Our previous publication demonstrated 33.3% of ED encounters with out-of-range factor replacement. Following several interventions, the cumulative rate of encounters with out-of-range dosing decreased to 18%. Overall, there was an increase in the mean percent of encounters receiving optimal factor dosing for both HA/HB compared to baseline (82.2% vs. 71.1%), though this was not a statistically significant difference. CONCLUSION: Despite implementation of multiple interventions, out-of-range factor dosing continues to occur. Our team plans to reinstate simulation center education for ED staff and continue education efforts of pharmacists and hematology trainees with the goal of further reducing out-of-range dosing in our ED.
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Hemofilia A , Mejoramiento de la Calidad , Niño , Humanos , Factores de Coagulación Sanguínea/uso terapéutico , Hemofilia A/tratamiento farmacológico , Hemorragia/etiología , Servicio de Urgencia en HospitalRESUMEN
Background: Children receiving hematopoietic stem cell transplantation (HCT) often experience an unfortunate sequalae of negative effects including pain, deconditioning, and anxiety. Massage therapy (MT) has demonstrated effective non-pharmacological management of fatigue, pain, and anxiety in patients undergoing cancer treatment. Existing studies have been limited by the lack of available MT-specific outcome measures to track responses to interventions. Purpose: This study aimed to describe the creation of a novel MT-specific outcome measure to be utilized in the pediatric acute-care setting and establish construct validity for this measure to assess clinical effectiveness of MT interventions. Setting: An oncology ward at a large pediatric tertiary medical center in the United States. Participants: A total of 58 children and young adults undergoing HCT. Research Design: Retrospective Cohort Study. Intervention: A panel of massage therapists created a novel outcome measure, OMPREP, for use in MT sessions and performed a literature review to ensure face validity of the tool. This outcome measure was administered to patients and data were collected retrospectively to assess construct validity. Results: A total of 1,333 MT sessions were completed (80.7% completion rate) with the novel OMPREP outcome measure utilized on 100% of visits. Mean engagement (p<.001), response (p<.001), and pain (p<.001) scores were all significantly greater at evaluation and discharge compared to the lowest observed scores post-HCT. Conclusion: The novel MT-specific outcome measure, OMPREP, was feasible and demonstrated construct validity when implemented in a pediatric acute-care setting by massage therapists. This new tool may offer a quantitative measure of MT-interventions and assist in tracking patient outcomes.
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Pregnancy and sickle cell disease (SCD) both individually carry a risk of thromboembolism (TE). Pregnancy in people with SCD may further enhance the prothrombotic effect of the underlying disease. The objectives of this study were to determine the rate and risk factors for arterial and venous thrombosis in pregnant people with SCD. Administrative claims data from the United States Centers for Medicare and Medicaid Service Analytic eXtract from 2006 to 2018 were used. The study population included people with SCD from the start of their first identified pregnancy until 1 year postpartum and a control cohort of pregnant people without SCD of similar age and race. Outcomes of interest were identified with ICD-9 or 10 codes. Logistic regression analyses were used to analyze risk factors. We identified infant deliveries in 6388 unique people with SCD and 17 110 controls. A total of 720 venous thromboembolism (11.3%) and 335 arterial TE (5.2%) were observed in people with SCD compared to 202 (1.2%) and 95 (0.6%) in controls. People with SCD had an 8-11 times higher odds of TE compared to controls (p < .001). Within the SCD cohort, age, hemoglobin SS (HbSS) genotype, hypertension, and history of thrombosis were identified as independent risk factors for pregnancy-related TE. Pregnancy-specific factors (pre-eclampsia, eclampsia, multigestational pregnancy) were not associated with TE. In conclusion, the risk of pregnancy-related TE is considerably higher in people with SCD compared with controls without SCD. Hence, people with SCD, particularly those with multiple risk factors may be candidates for thromboprophylaxis during pregnancy and the postpartum period.
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Anemia de Células Falciformes , Tromboembolia Venosa , Anciano , Embarazo , Humanos , Femenino , Estados Unidos/epidemiología , Medicaid , Anticoagulantes , Tromboembolia Venosa/epidemiología , Tromboembolia Venosa/etiología , Medicare , Anemia de Células Falciformes/complicaciones , Anemia de Células Falciformes/epidemiologíaRESUMEN
Patients with sickle cell disease (SCD) are at a risk of thromboembolism (TE), and use of hormonal contraception can further increase that risk. This study aims to assess patterns of hormonal contraceptive use and compare risk of contraception-related TE between combined hormonal contraceptives (CHCs) and progestin-only contraceptives (POCs). Patients with SCD aged between 12 and 44 years with a new prescription of a hormonal contraceptive in the Centers for Medicare and Medicaid Services Medicaid Analytic eXtract database (2006-2018) were followed up to 1 year. We identified 7173 new users: 44.6% initiated CHC and 55.4% initiated POC. Combined oral contraceptive pills (OCPs; 36.5%) and progestin-only depot medroxyprogesterone acetate (33.9%) were the most frequently prescribed agents. A total of 1.8% of contraception users had a new diagnosis of TE within 1 year of the first identified contraception prescription. There were no significant differences in TE event rates between CHC and POC users (17.2 and 24.7 events per 1000 person-years, respectively). In patients prescribed OCP, there were no differences in TE event rates based on estrogen dose or progestin generation. Transdermal patch had a 2.4-fold increased risk of TE as compared with that of OCP. Although limited by the retrospective study design and use of administrative claims data, this study found no significant differences in TE rates between new users of CHC and POC in patients with SCD. Careful evaluation of underlying TE risk factors should be considered for each patient with SCD before initiation of hormonal contraception.
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Anemia de Células Falciformes , Tromboembolia , Estados Unidos/epidemiología , Femenino , Humanos , Anciano , Niño , Adolescente , Adulto Joven , Adulto , Progestinas/efectos adversos , Anticoncepción Hormonal , Anticonceptivos Hormonales Orales/efectos adversos , Estudios Retrospectivos , Medicare , Tromboembolia/epidemiología , Tromboembolia/etiología , Anemia de Células Falciformes/complicaciones , Anemia de Células Falciformes/tratamiento farmacológico , Anemia de Células Falciformes/epidemiologíaRESUMEN
There is a lack of data on the safety and efficacy of peritoneal drain (PD) and chest tube (CT) in the management of effusions in stem cell transplant recipients with veno-occlusive disease (VOD). In this retrospective pediatric study, clinical outcomes and health resource utilization (HRU) were compared in 32 patients with VOD who had a PD (PD+) post-HCT versus 27 patients who did not (PD-). Nine patients also had a CT (7 PD+ and 2 PD-). PD + patients were more likely than PD-patients to have received myeloablative conditioning (100% vs. 85.2%; p = 0.04) and have severe or very severe VOD (100% vs. 56% p < 0.01). Mechanical obstruction (38%) and hypotension (38%) were common complications, and 13% developed peritonitis. While the frequencies of cardiac dysfunction and acute kidney injury were comparable between both groups, respiratory support and its median duration were higher in PD + patients. The hospital and intensive care unit length of stay, albumin use, and the duration of defibrotide and albumin therapy was significantly longer in PD + patients. At a median follow-up of 1.04 years (range:0.03-14.6), the 2-year overall survival was similar in both groups (53.8% vs. 51.5%; p = 0.73). Although PD use was similar between 1995 and 2007 vs. 2008-2021; (47% vs. 58%; p = 0.65), day+100 mortality was improved in recent years (53.3% vs. 17.8%; p = 0.01), coinciding with the use of defibrotide (0% vs. 84%; p < 0.01). PD in pediatric patients with VOD post-HCT, although associated with increased HRU, was safe when clinically indicated and did not adversely impact clinical outcomes.
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Trasplante de Células Madre Hematopoyéticas , Enfermedad Veno-Oclusiva Hepática , Niño , Humanos , Trasplante de Células Madre Hematopoyéticas/efectos adversos , Estudios Retrospectivos , Receptores de Trasplantes , Enfermedad Veno-Oclusiva Hepática/tratamiento farmacológico , PolidesoxirribonucleótidosRESUMEN
The study was conducted to examine prevalence of pulmonary embolism in children with sickle cell disease (SCD) and identify potential risk factors associated with pulmonary embolism in a single tertiary paediatric centre. Children with SCD between 0 and 21âyears of age from January 2010 to January 2021 were included. Pulmonary embolism was initially identified using International Classification of Diseases (ICD)-9 or 10 codes and confirmed with manual chart review of identified cases. Logistic regression analysis was performed to assess association between SCD specific and general thrombotic risk factors and pulmonary embolism. We identified 492 unique patients with SCD with a median age of 11âyears (interquartile range: 4-18). A total of eight (1.6%) patients developed a pulmonary embolism. Patients with pulmonary embolism were significantly older (median, interquartile range: 20.5, 14-21âyears) than patients without pulmonary embolism (median, interquartile range: 10, 4-17âyears). Central nervous system (CNS) vasculopathy and erythrocytapheresis were significantly associated with pulmonary embolism on univariable logistic regression analysis. A previous diagnosis of deep vein thrombosis (DVT) was significantly more common among patients with pulmonary embolism than among those without pulmonary embolism (50 vs. 5.2%; P â<â0.0001). Prevalence of pulmonary embolism in children with SCD was high. Risk factors associated with pulmonary embolism in this study such as CNS vasculopathy or erythrocytapheresis could suggest that the risk for pulmonary embolism in SCD may be related to the severity of disease state. Future studies are needed on pulmonary embolism prevention strategies.
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Anemia de Células Falciformes , Embolia Pulmonar , Humanos , Niño , Estudios Retrospectivos , Prevalencia , Embolia Pulmonar/complicaciones , Embolia Pulmonar/epidemiología , Anemia de Células Falciformes/complicaciones , Anemia de Células Falciformes/epidemiología , Factores de RiesgoRESUMEN
BACKGROUND: Evidence-based anticoagulation programs usually serve a local, adult patient population. Here we report outcomes for a regional combined pediatric-adult program. AIMS: The aims of this study were: (1) Compare the pre- vs. post-implementation quality of therapy (% time in therapeutic range (%TTR) and compliance). (2) Assess anticoagulant-relevant outcomes (bleeding and thrombotic complications). METHODS: Data were collected for the years 2014-2019. Rosendaal linear interpolation was used to calculate %TTR. Bleeding complications were categorized using ISTH-SSC standard nomenclature and new thrombotic events were reviewed. RESULTS: The patients were divided into a long-term warfarin group (N = 308), 80.2% of whom had cardiac-related therapeutic indications (median age 24y), and a second group (N = 114) comprised of short-term and non-warfarin long-term anticoagulation (median age 16y). Median %TTR for those on long-term warfarin was 78.9%. The incidence of major and clinically relevant non-major bleeding events was 1.65 and 2.43 /100 person-years of warfarin use, respectively. Thromboembolism (TE) incidence was 0.78/100 patient-years of warfarin use. Neither bleeding nor thrombosis was associated with %TTR (p = 0.48). Anticoagulant indication was the only variable associated with bleeding risk (p = 0.005). The second group had no on-therapy TE events but 7.9% experienced bleeding. Complete data were available for a randomly sampled pre-program warfarin group (N = 26). Median %TTR improved from 17.5 to 87% pre- vs. post-implementation. Similarly, compliance (defined as ≥ 1 INR/month) improved by 34.3%. CONCLUSIONS: In conclusion, this program significantly improved and sustained %TTR and compliance. The lack of association between bleeding and thrombosis events and %TTR may be related to the high median %TTR (> 70%) achieved by this approach.
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Tromboembolia , Trombosis , Humanos , Niño , Adulto , Adulto Joven , Adolescente , Relación Normalizada Internacional , Warfarina/efectos adversos , Anticoagulantes/efectos adversos , Coagulación Sanguínea , Hemorragia/inducido químicamente , Hemorragia/tratamiento farmacológico , Tromboembolia/tratamiento farmacológico , Trombosis/tratamiento farmacológico , Resultado del TratamientoRESUMEN
OBJECTIVE: To describe disease outcomes including overall survival and relapse patterns by subgroup in young pediatric patients treated for medulloblastoma with a radiation-sparing approach. METHODS: Retrospective analysis of clinical outcomes includes treatment, relapse, and salvage therapy and late effects in children treated for medulloblastoma with a radiation-sparing approach at British Columbia Children's Hospital (BCCH) between 2000 and 2020. RESULTS: There were 30 patients (median age 2.8 years, 60% male) treated for medulloblastoma with a radiation-sparing approach at BCCH. Subgroups included Sonic Hedgehog (SHH) (n = 14), group 3 (n = 7), group 4 (n = 6), and indeterminate status (n = 3). Three- and 5-year event-free survival (EFS) were 49.0% (30.2-65.4%) and 42.0% (24.2-58.9%) and overall survival (OS) 66.0% (95% CI 46.0-80.1%) and 62.5% (95% CI 42.5 and 77.2%), respectively, with a median follow-up of 9.5 years. Relapse occurred in 12/25 patients following a complete response, of whom six (group 4: n = 4; group 3: n = 1; unknown: n = 1) were successfully salvaged with craniospinal axis (CSA) RT and remain alive at a median follow-up of 7 years. Disease/treatment-related morbidity included endocrinopathies (n = 8), hearing loss n = 16), and neurocognitive abnormalities (n = 9). CONCLUSIONS: This radiation sparing treatment approach for young patients with medulloblastoma resulted in a durable cure in most patients with SHH subgroup medulloblastoma. In those patients with groups 3 and 4 medulloblastoma, relapse rates were high; however, most group 4 patients were salvaged with RT.
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Neoplasias Cerebelosas , Meduloblastoma , Niño , Humanos , Masculino , Preescolar , Femenino , Estudios Retrospectivos , Proteínas Hedgehog , Meduloblastoma/tratamiento farmacológico , Neoplasias Cerebelosas/tratamiento farmacológico , RecurrenciaRESUMEN
BACKGROUND: Despite needing to be informed about sickle cell trait (SCT) status to make informed reproductive decisions, more than 80% of adults with SCT, including parents of children with SCT who have a high prevalence of SCT, do not know their status. PROCEDURE: This was a prospective study of parents who received SCT telephone education from the state department of health and then completed SCTaware, a videoconference-administered SCT education program. The objectives were to evaluate knowledge after telephone education and explore if SCTaware closes knowledge gaps. Participants completed a demographic survey, a health literacy assessment, and reported their SCT status. They completed the Sickle Cell Trait Knowledge Assessment before receiving SCTaware, immediately after, and at follow-up visits; high knowledge was a score of 75% or higher correct. RESULTS: SCTaware and the initial surveys were completed by 61 parents; 45 completed the 6-month surveys. Only 43% of participants had high SCT knowledge after telephone education; 92% achieved high knowledge immediately after, and 84% continued with high knowledge at 6 months. Most parents reported they were aware of their SCT status after telephone education; 12 changed their response after receiving SCTaware. CONCLUSIONS: Our findings suggest that over half of parents have low SCT knowledge following telephone education, and many may be unaware of their status. SCTaware closes knowledge gaps, leads to high sustained knowledge, and is a potentially scalable tool. Future studies should refine SCTaware and determine if parents use their knowledge to inform their children and reproductive decisions.
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Anemia de Células Falciformes , Rasgo Drepanocítico , Adulto , Humanos , Niño , Rasgo Drepanocítico/epidemiología , Estudios Prospectivos , Conocimientos, Actitudes y Práctica en Salud , PadresRESUMEN
Patients with sickle cell disease (SCD) are predisposed to a hypercoagulable state. Despite the increased risk of venous thromboembolism in the SCD population, there is limited evidence available to guide thromboprophylaxis (TP) practices in these patients. This study aimed to assess the use of pharmacologic and nonpharmacologic TP in adolescent patients with SCD using the Pediatric Health Information System (PHIS). We hypothesized that TP was increasingly used in hospitalized adolescent patients with SCD. The study included patients with SCD aged 13 to 21 years, admitted to a PHIS hospital between 1 January, 2010, and 30 June, 2021. A total of 7202 unique patients consisting of 34 094 unique admissions were included for analyses. Pharmacologic or mechanical TP was used in 2600 (7.6%) admissions, with 3.6% of admissions (n = 1225) receiving pharmacologic prophylaxis and 4.3% (n = 1474) receiving mechanical prophylaxis. Pharmacologic TP increased in use from 1.3% of admissions in 2010 to 14.4% in the first half of 2021. Enoxaparin was the most commonly prescribed anticoagulant, used in 87% of admissions in which pharmacologic TP was used. The use of prophylactic direct oral anticoagulants was first documented in 2018 and increased to 25% of admissions with pharmacologic TP by 2021. This study demonstrates a steady increase in TP use in adolescent patients with SCD admitted to the hospital. Prospective cohort studies are needed to determine VTE risk factors in adolescents and children with SCD and the efficacy and safety of prophylactic regimens.
Asunto(s)
Anemia de Células Falciformes , Tromboembolia Venosa , Humanos , Adolescente , Niño , Anticoagulantes/uso terapéutico , Tromboembolia Venosa/etiología , Tromboembolia Venosa/prevención & control , Tromboembolia Venosa/epidemiología , Pacientes Internos , Estudios Prospectivos , Anemia de Células Falciformes/complicaciones , Anemia de Células Falciformes/tratamiento farmacológicoRESUMEN
Comorbid pulmonary complications in people with sickle cell disease (pwSCD) are associated with high rates of morbidity and mortality, and poor access to care contributes to poor outcomes among this particularly high-risk pwSCD. Our purpose was to describe the population served and the resources required for hematology, pulmonary, nursing, respiratory therapy, social work, genetics, psychology, and school liaison providers to see these patients in an integrated clinic. We abstracted demographic, medication, clinical, and diagnostics data of the pwSCD seen at least once in this clinic from February 1, 2014 to December 10, 2020 from the electronic medical record and identified 145 unique pwSCD. Abnormal lung function and bronchodilator responsiveness were detected in 31% and 42% of participants respectively. Sleep abnormalities were found in over two-thirds of those screened and 65% had ≥1 previous acute chest syndrome episode. This clinic also allowed for direct provider communication and required relatively limited resources to serve a large number of severely affected pwSCD. Given the degree of abnormal respiratory variables detected and the limited resources required to implement this model, studies are warranted to evaluate whether it has the potential to improve outcomes in high-risk populations.