RESUMO
ABSTRACT: In the setting of a learning collaborative, we conducted an international multicenter phase 2 clinical trial testing the hypothesis that nonmyeloablative-related haploidentical bone marrow transplant (BMT) with thiotepa and posttransplant cyclophosphamide (PTCy) will result in 2-year event-free survival (no graft failure or death) of at least 80%. A total of 70 participants were evaluable based on the conditioning protocol. Graft failure occurred in 8 of 70 (11.4%) and only in participants aged <18 years; all had autologous reconstitution. After a median follow-up of 2.4 years, the 2-year Kaplan-Meier-based probability of event-free survival was 82.6%. The 2-year overall survival was 94.1%, with no difference between children and adult participants. After excluding participants with graft failure (n = 8), participants with engraftment had median whole blood donor chimerism values at days +180 and +365 after transplant of 100% (n = 58), respectively, and 96.6% (57/59) were off immunosuppression 1 year after transplant. The 1-year grade 3 to 4 acute graft-versus-host disease (GVHD) rate was 10%, and the 2-year moderate-severe chronic GVHD rate was 10%. Five participants (7.1%) died from infectious complications. We demonstrate that nonmyeloablative haploidentical BMT with thiotepa and PTCy is a readily available curative therapy for most adults, even those with organ damage, compared to the more expensive myeloablative gene therapy and gene editing. Additional strategies are required for children to decrease graft failure rates. The trial was registered at www.clinicaltrials.gov as #NCT01850108.
Assuntos
Anemia Falciforme , Transplante de Medula Óssea , Doença Enxerto-Hospedeiro , Transplante Haploidêntico , Humanos , Transplante de Medula Óssea/métodos , Transplante de Medula Óssea/efeitos adversos , Masculino , Feminino , Criança , Adolescente , Adulto , Anemia Falciforme/terapia , Doença Enxerto-Hospedeiro/etiologia , Doença Enxerto-Hospedeiro/prevenção & controle , Transplante Haploidêntico/métodos , Pré-Escolar , Adulto Jovem , Ciclofosfamida/uso terapêutico , Ciclofosfamida/administração & dosagem , Condicionamento Pré-Transplante/métodos , Pessoa de Meia-Idade , Tiotepa/administração & dosagem , Tiotepa/uso terapêuticoRESUMO
Oral iron supplementation in iron deficient children with sickle cell anemia and normal transcranial Doppler ultrasound (TCD) velocities does not reduce arterial flow in the middle cerebral artery.
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Anemia Falciforme , Acidente Vascular Cerebral , Criança , Humanos , Velocidade do Fluxo Sanguíneo , Anemia Falciforme/complicações , Anemia Falciforme/diagnóstico por imagem , Ultrassonografia Doppler Transcraniana , Circulação CerebrovascularRESUMO
Children with sickle cell anemia (SCA) living in Nigeria are at an increased risk of malnutrition, which contributes to increased morbidity and mortality. However, evidence-based guidelines for managing malnutrition in children with SCA are lacking. To address this gap, we conducted a multicenter, randomized controlled feasibility trial to assess the feasibility and safety of treating children with SCA aged from 5 to 12 years and having uncomplicated severe acute malnutrition (body mass index z score of <-3.0). Children with SCA and uncomplicated severe acute malnutrition were randomly allocated to receive supplemental ready-to-use therapeutic food (RUTF) with or without moderate-dose hydroxyurea therapy (20 mg/kg per day). Over a 6-month enrollment period, 3190 children aged from 5 to 12 years with SCA were evaluated for eligibility, and 110 of 111 children who were eligible were enrolled. During the 12-week trial, no participants withdrew or missed visits. One participant died of unrelated causes. Adherence was high for hydroxyurea (94%, based on pill counts) and RUTF (100%, based on the number of empty sachets returned). No refeeding syndrome event or hydroxyurea-related myelosuppression occurred. At the end of the trial, the mean change in body mass index z score was 0.49 (standard deviation = 0.53), and 39% of participants improved their body mass index z score to ≥-3.0. Our findings demonstrate the feasibility, safety, and potential of outpatient treatment for uncomplicated severe acute malnutrition in children with SCA aged from 5 to 12 years in a low-resource setting. However, RUTF sharing with household and community members potentially confounded the response to malnutrition treatment. This trial was registered at clinicaltrials.gov as #NCT03634488.
Assuntos
Anemia Falciforme , Desnutrição , Desnutrição Aguda Grave , Humanos , Criança , Nigéria/epidemiologia , Hidroxiureia/efeitos adversos , Estudos de Viabilidade , Desnutrição Aguda Grave/complicações , Desnutrição/etiologia , Anemia Falciforme/complicações , Anemia Falciforme/tratamento farmacológicoRESUMO
We tested the hypothesis that fixed oral moderate-dose hydroxyurea (20 mg/kg per day) for initial treatment of secondary stroke prevention results in an 80% relative risk reduction of stroke or death when compared with fixed oral low-dose hydroxyurea (10 mg/kg per day) in a phase 3 double-blind, parallel-group, randomized controlled trial in children with sickle cell anemia (SCA) living in Nigeria. A total of 101 participants were randomly allocated to low-dose (n = 49) and moderate-dose (n = 52) hydroxyurea treatment groups. The median participant follow-up was 1.6 years (interquartile range, 1.0-2.3), with a planned minimum follow-up of 3.0 years. A total of 6 recurrent strokes and 2 deaths vs 5 recurrent strokes and 3 deaths occurred in the low- and moderate-dose groups, respectively. The incidence rate ratio (IRR) of the primary outcome measure of stroke or death in the low- and moderate-dose hydroxyurea treatment groups was 0.98 (95% confidence interval [CI], 0.32-3.00; P = .97). The trial was stopped early owing to no clinical difference in the incidence rates of the primary outcome measure. The incidence rates of recurrent strokes were 7.1 and 6.0 per 100 person-years in the low- and moderate-dose groups, respectively, (IRR, 1.18; 95% CI, 0.30-4.88; P = .74). As a measure of adherence to the oral hydroxyurea therapy, the median percent of returned pills was 3.0% and 2.6% in the low- and moderate-dose groups, respectively. No participant had hydroxyurea therapy stopped for myelosuppression. For children with SCA in low-income settings without access to regular blood transfusion therapy, initial low-dose hydroxyurea is a minimum known efficacious dose for secondary stroke prevention.
Assuntos
Anemia Falciforme , Acidente Vascular Cerebral , Criança , Humanos , Hidroxiureia/uso terapêutico , Antidrepanocíticos/uso terapêutico , Nigéria , Anemia Falciforme/complicações , Acidente Vascular Cerebral/etiologia , Prevenção Secundária/métodosRESUMO
The Food and Drug Administration requires contemporaneous controls to compare clinical outcomes for participants receiving experimental gene therapy or gene editing clinical trials. However, developing a contemporaneous cohort of rare diseases requires multiple person-hours. In a single referral center for sickle cell disease, we tested the hypothesis that we could create an automated contemporaneous cohort of children and adults with sickle cell anemia (SCA) to predict mortality. Data were obtained between 1 January 2004 and 30 April 2021. We identified 419 individuals with SCA with consistent medical care defined as followed continuously for >0.5 years with no visit gaps >3.0 years. The median age was 10.2 years (IQR, 1-24 years), with a median follow-up of 7.4 years (IQR, 3.6-13.5 years) and 47 deaths. A total of 98% (274 of 277) of the children remained alive at 18 years of age, and 34.3% (94 of 274) of those children were followed into adulthood. For adults, the median age of survival was 49.3 years. Treatment groups were mutually exclusive and in a hierarchical order: hematopoietic stem cell transplant (n = 22)>regular blood transfusion for at least 2 years (n = 56)>hydroxyurea for at least 1 year (n = 243)>no disease-modifying therapy (n = 98). Compared to those receiving no disease-modifying treatment, those treated with hydroxyurea therapy had a significantly lower hazard of mortality (hazard ratio = 0.38; P = 0.016), but no statistical difference for those receiving regular blood transfusions compared to no disease-modifying therapy (hazard ratio = 0.71; P = 0.440). An automated contemporaneous SCA cohort can be generated to estimate mortality in children and adults with SCA.
Assuntos
Anemia Falciforme , Acidente Vascular Cerebral , Estados Unidos , Criança , Adulto , Humanos , Pessoa de Meia-Idade , Hidroxiureia/uso terapêutico , Antidrepanocíticos/uso terapêutico , Acidente Vascular Cerebral/tratamento farmacológico , Anemia Falciforme/complicações , Anemia Falciforme/terapia , Transfusão de SangueRESUMO
BACKGROUND: In high-income countries, standard care for primary stroke prevention in children with sickle cell anaemia and abnormal transcranial Doppler velocities results in a 92% relative risk reduction of strokes but mandates initial monthly blood transfusion. In Africa, where regular blood transfusion is not feasible for most children, we tested the hypothesis that initial moderate-dose compared with low-dose hydroxyurea decreases the incidence of strokes for children with abnormal transcranial Doppler velocities. METHODS: SPRING is a double-blind, parallel-group, randomised, controlled, phase 3 trial of children aged 5-12 years with sickle cell anaemia with abnormal transcranial Doppler velocities conducted at three teaching hospitals in Nigeria. For randomisation, we used a permuted block allocation scheme with block sizes of four, stratified by sex and site. Allocation was concealed from all but the pharmacists and statisticians. Participants were assigned in a 1:1 ratio to low-dose (10 mg/kg per day) or moderate-dose (20 mg/kg per day) oral hydroxyurea taken once daily with monthly clinical evaluation and laboratory monitoring. The primary outcome was initial stroke or transient ischaemic attack, centrally adjudicated. The secondary outcome was all-cause hospitalisation. We used the intention-to-treat population for data analysis. The trial was stopped early for futility after a planned minimum follow-up of 3·0 years to follow-up for participants. This trial was registered with ClinicalTrials.gov, number NCT02560935. FINDINGS: Between Aug 2, 2016, and June 14, 2018, 220 participants (median age 7·2 years [IQR 5·5-8·9]; 114 [52%] female) were randomly allocated and followed for a median of 2·4 years (IQR 2·0-2·8). All participants were Nigerian and were from the following ethnic groups: 179 (82%) people were Hausa, 25 (11%) were Fulani, and 16 (7%) identified as another ethnicity. In the low-dose hydroxyurea group, three (3%) of 109 participants had strokes, with an incidence rate of 1·19 per 100 person-years and in the moderate-dose hydroxyurea group five (5%) of 111 had strokes with an incidence rate of 1·92 per 100 person-years (incidence rate ratio 0·62 [95% CI 0·10-3·20], p=0·77). The incidence rate ratio of hospitalisation for any reason was 1·71 (95% CI 1·15-2·57, p=0·0071), with higher incidence rates per 100 person-years in the low-dose group versus the moderate-dose group (27·43 vs 16·08). No participant had hydroxyurea treatment stopped for myelosuppression. INTERPRETATION: Compared with low-dose hydroxyurea therapy, participants treated with moderate-dose hydroxyurea had no difference in the stroke incidence rate. However, secondary analyses suggest that the moderate-dose group could lower incidence rates for all-cause hospitalisations. These findings provide an evidence-based guideline for the use of low-dose hydroxyurea therapy for children with sickle cell anaemia at risk of stroke. FUNDING: National Institute of Neurological Disorders and Stroke.
Assuntos
Anemia Falciforme , Acidente Vascular Cerebral , Anemia Falciforme/complicações , Anemia Falciforme/tratamento farmacológico , Antidrepanocíticos/uso terapêutico , Pré-Escolar , Método Duplo-Cego , Feminino , Humanos , Hidroxiureia/uso terapêutico , Nigéria , Acidente Vascular Cerebral/etiologia , Acidente Vascular Cerebral/prevenção & controleRESUMO
Strokes in children with sickle cell anemia (SCA) are associated with significant morbidity and premature death. Primary stroke prevention in children with SCA involves screening for abnormal transcranial Doppler (TCD) velocity coupled with regular blood transfusion therapy for children with abnormal velocities, for at least one year. However, in Africa, where the majority of children with SCA live, regular blood transfusions are not feasible due to inadequate supply of safe blood, cost, and the reluctance of caregivers to accept transfusion therapy for their children. We describe the Primary Prevention of Stroke in Children with Sickle Cell Disease in Nigeria Trial [StrokePreventioninNigeria (SPRING) trial, NCT02560935], a three-center double-blinded randomized controlled Phase III clinical trial to 1) determine the efficacy of moderate fixed-dose (20 mg/kg/day) versus low fixed-dose (10 mg/kg/day) hydroxyurea therapy for primary stroke prevention; 2) determine the efficacy of moderate fixed-dose hydroxyurea for decreasing the incidence of all cause-hospitalization (pain, acute chest syndrome, infection, other) compared to low fixed-dose hydroxyurea. We will test the primary hypothesis that there will be a 66% relative risk reduction of strokes in children with SCA and abnormal TCD measurements, randomly allocated, for a minimum of three years to receive moderate fixed-dose versus low fixed-dose hydroxyurea (total n = 220). The results of this trial will advance the care of children with SCA in sub-Saharan Africa, while improving research capacity for future studies to prevent strokes in children with SCA.
Assuntos
Anemia Falciforme/complicações , Acidente Vascular Cerebral/prevenção & controle , África Subsaariana , Feminino , Humanos , Masculino , Acidente Vascular Cerebral/epidemiologiaRESUMO
BACKGROUND: Children with sickle cell disease (SCD) are at risk for cognitive deficits. Limited data describe whether comprehensive cognitive evaluation improves education resources and whether caregivers perceive the assessment as beneficial. We tested our two hypotheses: (a) an integrated comprehensive cognitive evaluation program in children with SCD results in increased special education services allocation; and (b) caregivers will value comprehensive cognitive evaluation services provided. PROCEDURE: In a tertiary care medical facility, as part of quality improvement project, in a before-and-after evaluation between March 2011 and July 2014, we examined the impact of targeted comprehensive cognitive evaluation on change in special education services. We also evaluated the caregiver's perception regarding the utility of the provided services. RESULTS: A total of 21% (42 of 196) students (median age 11 years, range 3-18) with SCD were referred for cognitive assessment due to overt stroke (n = 11), silent stroke (n = 14), or concerns about cognitive or academic functioning without evidence of strokes (n = 17). At baseline, 45.2% received special education services and after the comprehensive cognitive evaluation 86.7% received special education services (P < 0.001). Among 33 caregivers who completed the survey, 97% reported that the assessment was helpful and 60% indicated that assessment led to beneficial changes for their children at school. CONCLUSION: Education advocacy coupled with comprehensive cognitive assessment in students with SCD improved access to special education services, and caregivers uniformly endorse this service as having added value.
Assuntos
Anemia Falciforme/psicologia , Anemia Falciforme/terapia , Cuidadores/educação , Cognição , Educação Inclusiva/normas , Recursos em Saúde/estatística & dados numéricos , Educação de Pacientes como Assunto/normas , Adolescente , Adulto , Criança , Pré-Escolar , Aconselhamento , Feminino , Seguimentos , Humanos , Masculino , Prognóstico , Avaliação de Programas e Projetos de Saúde , Adulto JovemRESUMO
BACKGROUND: Based on the presumed clinical similarity between the two most severe sickle cell disease (SCD) genotypes, hemoglobin (Hb) Sß0 thalassemia and HbSS, randomized controlled trials (RCTs) have included both genotypes. Our group has demonstrated that healthcare providers inadequately distinguish the two diagnoses through clinical and laboratory parameters. PROCEDURE: In a retrospective cohort study utilizing data from the Silent Cerebral Infarct Transfusion trial (NCT00072761), we tested the hypothesis that children with genotypic diagnoses of HbSß0 thalassemia, when compared to HbSS, have significantly different rates of SCD comorbidities. Exclusion criteria included those with previous overt stroke or treatment with hydroxyurea or regular blood transfusion therapy. RESULTS: Among children with HbSß0 thalassemia (n = 22) and HbSS (n = 786), the mean hemoglobin was higher in children with HbSß0 thalassemia (9.2 g/dl) compared to HbSS (8.1 g/dl, P < 0.001). In children with HbSß0 thalassemia, when compared to HbSS, the incidence rate of acute chest syndrome (ACS) was 3.0 and 14.4 events per 100 patient-years (P = 0.028), and mean transcranial Doppler (TCD) velocities were 112.6 and 135.6 cm/sec, respectively (P = 0.026). The number of children with HbSß0 thalassemia and HbSS with conditional TCD velocities were zero (0%) and 26 (4.9%), respectively (P = 1.00), and the number with silent cerebral infarcts were five (27.8%) and 209 (30.8%), respectively (P = 0.78). CONCLUSIONS: We have provided preliminary evidence that clinically relevant differences occur in ACS rates and TCD velocities between children with HbSß0 thalassemia and HbSS. Future SCD RCTs should consider balanced allocation of these SCD genotypes, particularly when ACS and abnormal TCD velocities are primary outcome measures.
Assuntos
Síndrome Torácica Aguda/epidemiologia , Anemia Falciforme/sangue , Anemia Falciforme/complicações , Talassemia/sangue , Talassemia/complicações , Síndrome Torácica Aguda/etiologia , Adolescente , Criança , Pré-Escolar , Feminino , Hemoglobina Falciforme , Humanos , Incidência , Masculino , Estudos RetrospectivosRESUMO
BACKGROUND: The relationship between silent cerebral infarcts (SCIs) and history of parvovirus B19 (B19V) has not been systematically evaluated. As an ancillary study from the Silent Cerebral Infarct Trial (SIT) (NCT00072761), we tested the hypothesis that a history of B19V infection is associated with an increased prevalence of SCIs in children with sickle cell anemia. PROCEDURE: We used a retrospective cross-sectional cohort study design; each participant underwent a brain magnetic resonance imaging (MRI) scan and medical record review for prior B19V infection (n = 958). RESULTS: SCI was present in 30% (287 of 958) of participants and 17% (165 of 958) had a history of B19V infection. Based on prior evidence that low baseline hemoglobin (Hgb) levels are associated with increased odds of SCI, Hgb levels were divided into tertiles (<7.6 g/dl, ≥7.6-≤8.5 g/dl, ≥8.6 g/dl) and multivariable analysis was used to determine the relationship between the joint effect of prior B19V infection, Hgb levels, and SCI. Prior B19V infection and the lowest Hgb tertile were associated with increased risk of SCI (odds ratio [OR] 2.12; 95% CI, 1.17-3.84; P = 0.013); no prior B19V infection and the highest Hgb tertile were associated with a decreased risk (OR 0.56; 95% CI, 0.38-0.84; P = 0.004). CONCLUSIONS: Efforts to decrease the incidence of B19V infection, such as the development of a B19V vaccine, may decrease SCI prevalence.
Assuntos
Infarto Cerebral , Eritema Infeccioso , Imageamento por Ressonância Magnética , Parvovirus B19 Humano , Infarto Cerebral/diagnóstico por imagem , Infarto Cerebral/epidemiologia , Infarto Cerebral/etiologia , Infarto Cerebral/virologia , Criança , Pré-Escolar , Eritema Infeccioso/complicações , Eritema Infeccioso/diagnóstico por imagem , Eritema Infeccioso/epidemiologia , Feminino , Humanos , Incidência , Masculino , Estudos Retrospectivos , Fatores de RiscoRESUMO
In a retrospective cohort study, we tested the hypothesis that when prescribing hydroxyurea (HU) to children with sickle cell anemia (SCA) to prevent vaso-occlusive events, there will be a secondary benefit of maintaining low transcranial Doppler (TCD) velocity, measured by imaging technique (TCDi). HU was prescribed for 90.9% (110 of 120) of children with SCA ≥5 years of age and followed for a median of 4.4 years, with 70% (n = 77) receiving at least one TCDi evaluation after starting HU. No child prescribed HU had a conditional or abnormal TCDi measurement. HU initiation for disease severity prevention decreases the prevalence of abnormal TCDi velocities.
Assuntos
Anemia Falciforme/tratamento farmacológico , Anemia Falciforme/fisiopatologia , Antidrepanocíticos/uso terapêutico , Circulação Cerebrovascular/efeitos dos fármacos , Hidroxiureia/uso terapêutico , Adolescente , Velocidade do Fluxo Sanguíneo , Criança , Pré-Escolar , Estudos de Coortes , Feminino , Humanos , Masculino , Estudos Retrospectivos , Ultrassonografia Doppler TranscranianaRESUMO
BACKGROUND: Young children with sickle cell disease (SCD) are at risk for cognitive delay. In addition to biologic risk factors associated with SCD, environmental factors contribute to cognitive dysfunction within this cohort. METHODS: We completed a single-arm, prospective cohort study. Children with SCD between the ages of 3 and 36 months and their caregivers were followed between October 2010 and December 2013. The aim was to describe the role of a home visitation model, the home environment, and socioeconomic status in the development of young children with SCD. Primary outcome measures were the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) and the Home Observation for Measurement of the Environment (HOME). We hypothesized that the home visitation model, Parents as Teachers® (PAT), would encourage positive parent-child interactions and improve cognitive outcomes. RESULTS: Thirty-five participants had at least two PAT visits and BSID-III assessments. Mean scores within all five subtests of the BSID-III improved between enrollment and exit, with significant changes within cognitive (P = 0.016) and expressive language (EL) domains (P = 0.002). Multivariate modeling found the HOME score associated with the exit results of the cognitive domain. CONCLUSION: We report longitudinal results of the first home visitation program within the early childhood SCD population and show significant improvement in cognitive and EL development. Additionally, home environment was a significant predictor of cognitive development. Randomized controlled trials to test the impact of interventions targeting the home environment are warranted for this vulnerable population.
Assuntos
Anemia Falciforme/fisiopatologia , Desenvolvimento Infantil , Pais , Educação de Pacientes como Assunto , Adulto , Criança , Cognição , Estudos de Coortes , Feminino , Humanos , Masculino , Projetos Piloto , Estudos Prospectivos , Classe SocialRESUMO
Although individuals with sickle cell anaemia (SCA) have elevated baseline inflammation and endothelial activation, the acute phase response to maximal exercise has not been evaluated among children with SCA. We measured the acute phase response to maximal exercise testing for soluble vascular cell adhesion molecule (sVCAM) as well as interleukin 6 (IL6), total white blood cell (WBC) count, C-reactive protein (CRP) and D-dimer in a cohort of children with SCA and matched controls at baseline, immediately after, and 30, 60 and 120 min following exercise. Despite higher baseline levels of all biomarkers except CRP, the acute phase response from baseline to immediately after exercise was significantly greater in subjects versus controls for CRP (2·1 vs. 0·2 mg/l, P = 0·02) and D-dimer (160 vs. 10 µg/l, P < 0·01) only. Similar between-group trends were observed over time for all biomarkers, including sVCAM, IL6, total WBC, CRP and D-dimer. Lower fitness, defined by peak oxygen consumption (VO2 ), was independently associated with greater acute phase responses to exercise for sVCAM. Our results suggest maximal exercise may not be associated with any greater escalation of endothelial activation or inflammation in SCA and provide preliminary biomarker evidence for the safety of brief, high-intensity physical exertion in children with SCA.
Assuntos
Reação de Fase Aguda/etiologia , Anemia Falciforme/fisiopatologia , Exercício Físico/fisiologia , Reação de Fase Aguda/metabolismo , Adolescente , Biomarcadores/metabolismo , Proteína C-Reativa/metabolismo , Estudos de Casos e Controles , Criança , Teste de Esforço , Produtos de Degradação da Fibrina e do Fibrinogênio/metabolismo , Humanos , Inflamação/fisiopatologia , Interleucina-6/metabolismo , Contagem de Leucócitos , Aptidão Física/fisiologia , Molécula 1 de Adesão de Célula Vascular/metabolismo , Adulto JovemRESUMO
BACKGROUND: Few studies have investigated factors influencing participation rates for minority children with a chronic disease in clinical trials. The Silent Cerebral Infarct Multi-Center Clinical (SIT) Trial provides an opportunity to study the impact of demographic and socio-economic factors on randomization in a clinical trial among Black children. Our primary objective was to characterize the factors associated with successful randomization of children with sickle cell disease (SCD) and silent cerebral infarct (SCI) in the SIT Trial after initial consent. PROCEDURE: Differences in socio-economic and demographic variables, family history and disease-related variables were determined between eligible participants who were successfully randomized and those who were not randomized following initial consent. Head of household educational level and family income were examined separately for US versus non-US sites. RESULTS: Of 1,176 children enrolled in the SIT Trial, 1,016 (86%) completed screening. Of 208 (20%) children with qualifying SCI on pre-randomization MRI, 196 (94%) were successfully randomized. There were no differences in socio-economic, demographic, or disease-related variables between children who were or were not randomized. Participants from non-US sites were more likely to be randomized (22% vs. 12%, P = 0.011); although, randomization by country was associated with neither head of household education nor family income. CONCLUSION: In the SIT Trial, acceptance of random allocation was not associated with socio-economic or demographic factors. Although these factors may represent barriers for some participants, they should not bias investigators caring for children with SCD in their approach to recruitment for clinical trial participation.
Assuntos
Anemia Falciforme/terapia , Transfusão de Sangue , Infarto Cerebral/terapia , Adolescente , Criança , Pré-Escolar , Demografia , Feminino , Seguimentos , Humanos , Masculino , Prognóstico , Fatores SocioeconômicosRESUMO
PURPOSE: Despite the recognized relevance of symptom burden in breast cancer, there has been limited exploration of whether an individual patient's assessment of the overall quality of care received might influence outcome. We therefore evaluated the relationship between patient-reported satisfaction with service quality and survival in breast cancer. METHODS: A random sample of 1,521 breast cancer patients treated at Cancer Treatment Centers of America. A questionnaire which covered several dimensions of patient satisfaction was administered. Items were measured on a seven-point Likert scale ranging from "completely dissatisfied" to "completely satisfied". Univariate and multivariate Cox regression was used to evaluate the association between patient satisfaction and survival. RESULTS: Of 1,521 patients, 836 were newly diagnosed, and 685 had previously been treated. A number of 409, 611, 323, and 178 patients had stage I, II, III, and IV disease, respectively. A total of 1,106 (72.7 %) patients were completely satisfied with the overall service quality, while 415 (27.3 %) were not. On univariate analysis, completely satisfied patients had a significantly lower risk of mortality compared to those not completely satisfied (HR = 0.62; 95 % CI 0.50-0.76; p < 0.001). On multivariate analysis, completely satisfied patients demonstrated significantly lower mortality (HR = 0.71; 95 % CI 0.57-0.87; p = 0.001) compared to those not completely satisfied. CONCLUSIONS: Patient satisfaction with service quality was an independent predictor of survival in breast cancer. Further exploration of a possible meaningful relationship between patient satisfaction with the care they receive and outcomes in breast cancer is indicated.
Assuntos
Neoplasias da Mama/psicologia , Neoplasias da Mama/terapia , Satisfação do Paciente/estatística & dados numéricos , Adulto , Idoso , Idoso de 80 Anos ou mais , Feminino , Humanos , Pessoa de Meia-Idade , Análise Multivariada , Modelos de Riscos Proporcionais , Inquéritos e Questionários , Resultado do TratamentoRESUMO
OBJECTIVE: To evaluate the relationship between self-reported satisfaction with service quality and overall survival in non-small cell lung cancer (NSCLC). DESIGN: A prospective cohort study. SETTING: Cancer Treatment Centers of America(®) from July 2007 and December 2010. PARTICIPANTS: Nine hundred and eighty-six returning NSCLC patients. INTERVENTION: Overall patient experience 'considering everything, how satisfied are you with your overall experience' was measured on a 7-point Likert scale ranging from 'completely dissatisfied' to 'completely satisfied.'. MAIN OUTCOME MEASURE: Patient survival was the primary end point. RESULTS: The response rate for this study was 69%. Six hundred patients were newly diagnosed, while 386 were previously treated. Four hundred sixty-nine were males, while 517 were females. 101, 59, 288 and 538 patients had stage I, II, III and IV disease, respectively. Mean age was 58.9 years. Six hundred and thirty (63.9%) patients had expired at the time of this analysis. Seven hundred and sixty-two (77.3%) patients were 'completely satisfied'. Median overall survival was 12.1 months (95% confidence interval (CI): 10.9-13.2 months). On univariate analysis, 'completely satisfied' patients had a significantly lower risk of mortality compared with those not 'completely satisfied' [hazard ratio (HR) = 0.70; 95% CI: 0.59-0.84; P < 0.001]. On multivariate analysis controlling for stage at diagnosis, prior treatment history, age and gender, 'completely satisfied' patients demonstrated significantly lower mortality (HR = 0.71; 95% CI: 0.60-0.85; P < 0.001) compared with those not 'completely satisfied'. CONCLUSIONS: Self-reported experience with service quality was an independent predictor of survival in NSCLC patients undergoing oncologic treatment, a novel finding in the literature. Based on these provocative findings, further exploration of this relationship is warranted in well-designed prospective studies.
Assuntos
Carcinoma Pulmonar de Células não Pequenas/terapia , Neoplasias Pulmonares/terapia , Satisfação do Paciente , Qualidade da Assistência à Saúde , Adulto , Idoso , Idoso de 80 Anos ou mais , Carcinoma Pulmonar de Células não Pequenas/diagnóstico , Carcinoma Pulmonar de Células não Pequenas/mortalidade , Carcinoma Pulmonar de Células não Pequenas/psicologia , Feminino , Humanos , Neoplasias Pulmonares/diagnóstico , Neoplasias Pulmonares/mortalidade , Neoplasias Pulmonares/psicologia , Masculino , Oncologia/normas , Oncologia/estatística & dados numéricos , Pessoa de Meia-Idade , Satisfação do Paciente/estatística & dados numéricos , Prognóstico , Estudos Prospectivos , Qualidade da Assistência à Saúde/estatística & dados numéricos , Inquéritos e Questionários , Análise de Sobrevida , Adulto JovemRESUMO
BACKGROUND: The contribution of environmental tobacco smoke (ETS) exposure to pulmonary morbidity in children with sickle cell anemia (SCA) is poorly understood. We tested the hypothesis that children with SCA and ETS exposure would have an increased prevalence of obstructive lung disease and respiratory symptoms compared with children with SCA and no ETS exposure. METHODS: Parent reports of ETS and respiratory symptom frequency were obtained for 245 children with SCA as part of a multicenter prospective cohort study. One hundred ninety-six children completed pulmonary function testing. Multivariable regression models were used to evaluate the associations between ETS exposure at different time points (prenatal, infant [birth to 2 years], preschool [2 years to first grade], and current) and lung function and respiratory symptoms. RESULTS: Among the 245 participants, a high prevalence of prior (44%) and current (29%) ETS exposure was reported. Of the 196 children who completed pulmonary function testing, those with parent-reported infant and current ETS exposure were more likely to have airway obstruction (defined as an FEV1/FVC ratio below the lower limit normal) compared with unexposed children (22.0% vs 3.1%, P < .001). Those with ETS exposure also had a lower forced expiratory flow, midexpiratory phase/FVC ratio (0.82 vs 0.97, P = .001) and were more likely to have evidence of bronchodilator responsiveness (23% vs 11%, P = .03). Current and prior ETS exposure and in utero smoke exposure were associated with increased frequency of respiratory symptoms. CONCLUSIONS: ETS exposure is associated with evidence of lower airway obstruction and increased respiratory symptoms in SCA.
Assuntos
Obstrução das Vias Respiratórias/etiologia , Anemia Falciforme/complicações , Poluição por Fumaça de Tabaco/efeitos adversos , Adolescente , Criança , Pré-Escolar , Estudos de Coortes , Estudos Transversais , Feminino , Humanos , Masculino , Estudos ProspectivosRESUMO
Despite the recognized relevance of symptom burden in colorectal cancer, there has been limited exploration of whether an individual patient's assessment of the overall quality-of-care received might influence outcome. We evaluated the relationship between patient-reported experience with service quality and survival in 702 returning colorectal cancer patients treated at our institution between July 2007 and December 2010. Overall patient experience "considering everything, how satisfied are you with your overall experience?" was measured on a 7-point Likert scale ranging from completely dissatisfied to completely satisfied. It was dichotomized into two categories: top box response (7) versus all others (1-6). Cox regression was used to evaluate the association between patient experience and survival. Of 702 patients, 506 were "completely satisfied" while 196 were not. On univariate analysis, "completely satisfied" patients had a significantly lower risk of mortality compared to those "not completely satisfied" (hazard ratio [HR] = 0.78; 95% confidence interval [CI]: 0.61-0.98; p = .04). Similarly, on multivariate analysis controlling for stage at diagnosis, treatment history, age, and gender, "completely satisfied" patients demonstrated significantly lower mortality (HR = 0.74; 95% CI: 0.58-0.95; p = .02). Patient experience with service quality was an independent predictor of survival in colorectal cancer, a novel finding in the literature.
Assuntos
Neoplasias Colorretais , Satisfação do Paciente , Qualidade da Assistência à Saúde , Sobrevida , Adulto , Idoso , Idoso de 80 Anos ou mais , Neoplasias Colorretais/mortalidade , Neoplasias Colorretais/patologia , Intervalos de Confiança , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Modelos de Riscos Proporcionais , Estados Unidos , Adulto JovemRESUMO
PURPOSE: Despite the recognized relevance of symptom burden in pancreatic cancer, there has been limited exploration of whether an individual patient's satisfaction with the overall quality of care received might influence outcome. We evaluated the relationship between patient satisfaction with health service quality and survival in patients with pancreatic cancer. PATIENTS AND METHODS: A random sample of 496 pancreatic cancer patients treated at Cancer Treatment Centers of America(®) (CTCA) between July 2007 and December 2010. A questionnaire that covered several dimensions of patient satisfaction was administered. Items were measured on a seven-point Likert scale ranging from "completely dissatisfied" to "completely satisfied." Patient survival was the primary end point. Cox regression was used to evaluate the association between patient satisfaction and survival. RESULTS: The response rate for this study was 72%. Of the 496 patients, 345 (69.6%) reported being "completely satisfied" with the care provided. Median overall survival was 7.9 months. On univariate analysis, patients reporting they were "completely satisfied" experienced superior survival compared with patients stating they were "not completely satisfied" (hazard ratio = 0.62; 95% confidence interval: 0.50-0.77; P < 0.001). On multivariate analysis controlling for stage at diagnosis, treatment history, and specific CTCA treatment center, "completely satisfied" patients demonstrated significantly lower mortality (hazard ratio = 0.63; 95% confidence interval: 0.51-0.79; P < 0.001). CONCLUSION: In this exploratory analysis, patient satisfaction with health service quality was an independent predictor of survival in pancreatic cancer. Further exploration of a possible meaningful relationship between patient satisfaction with the care they have received and outcome in this difficult malignancy is indicated.