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PURPOSE: Treatment-related pancreatitis (TRP) is a serious complication occurring in children with acute lymphoblastic leukemia (ALL). Those affected are at high risk for severe organ toxicity and treatment delays that can impact outcomes. TRP is associated with asparaginase, a standard therapeutic agent in childhood ALL. Native American ancestry, older age, high-risk leukemia, and increased use of asparaginase are linked to pancreatitis risk. However, dedicated genetic studies evaluating pancreatitis in childhood ALL include few Hispanics. Thus, the genetic basis for higher risk of pancreatitis among Hispanic children with ALL remains unknown. METHODS: Cases of children with ALL treated in from 1994 through 2013 were reviewed and identified 14, all Hispanic, who developed pancreatitis related to asparaginase therapy. Forty-six controls consisting of Hispanic children treated on the same regimens without pancreatitis were selected for comparison. Total DNA isolated from whole blood was used for targeted DNA sequencing of 23 selected genes, including genes associated with pancreatitis without ALL and genes involved in asparagine metabolism. RESULTS: Non-synonymous polymorphisms and frameshift deletions were detected in 15 genes. Most children with TRP had variants in ABAT, ASNS, and CFTR. Notably, children with TRP harbored many more CFTR variants (71.4%) compared with controls (39.1%). Among these, V470M (rs213950) was most frequent (OR 4.27, p = 0.025). CONCLUSIONS: This is the first study of genetic factors in treatment-related pancreatitis in Hispanic children with ALL. Identifying correlative variants in ethnically vulnerable populations may improve screening to identify which patients with ALL are at greatest risk for pancreatitis.
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Hispânico ou Latino/genética , Pancreatite/induzido quimicamente , Pancreatite/genética , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Leucemia-Linfoma Linfoblástico de Células Precursoras/genética , Adolescente , Asparaginase/administração & dosagem , Asparaginase/efeitos adversos , Carbono-Nitrogênio Ligases com Glutamina como Doadora de N-Amida/genética , Estudos de Casos e Controles , Criança , Pré-Escolar , Estudos de Coortes , Regulador de Condutância Transmembrana em Fibrose Cística/genética , Feminino , Marcadores Genéticos , Predisposição Genética para Doença , Humanos , Masculino , Pancreatite/terapiaRESUMO
BACKGROUND: Cancer-related sexual dysfunction has been reported among adolescents and young adults (AYAs); however, its prevalence over time has not been examined. This longitudinal study investigated sexual dysfunction in AYAs over the course of 2 years after the initial diagnosis. METHODS: Young adult patients (18-39 years old) completed the Medical Outcomes Study Sexual Functioning Scale within the first 4 months of their diagnosis (n = 123) and again 6 (n = 107) and 24 months later (n = 95). An ordered multinomial response model analyzed changes in the probability of reporting sexual dysfunction over time and the independent effects of demographic, clinical, and psychosocial variables. RESULTS: More than half of the participants reported sexual functioning to be problematic at each assessment. The probability of reporting sexual dysfunction increased over time (P < .01) and was greater for cancer patients who were female (P < .001), older (P < .01), married or in a committed relationship (P < .001), treated with chemotherapy (P < .05), and reporting comorbid psychological distress (P < .001) and lower social support (P < .05). For women, being in a relationship increased the likelihood of reporting sexual problems over time; for men, the likelihood of reporting sexual problems increased regardless of their relationship status. CONCLUSIONS: A substantial proportion of young adults report ongoing problems with sexual functioning in the first 2 years after their cancer diagnosis. These findings justify the need to evaluate and monitor sexual functioning throughout a continuum of care. Cancer 2018;124:398-405. © 2017 American Cancer Society.
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Neoplasias/psicologia , Comportamento Sexual/psicologia , Adolescente , Adulto , Feminino , Humanos , Estudos Longitudinais , Masculino , Probabilidade , Disfunções Sexuais Fisiológicas/epidemiologia , Disfunções Sexuais Psicogênicas/epidemiologia , Adulto JovemRESUMO
BACKGROUND: The objective of the current study was to examine social functioning among adolescents and young adults (AYAs) within the first 2 years after a cancer diagnosis and compare their scores with population norms and identify trajectories of social functioning over time and its correlates. METHODS: A multicenter, longitudinal study was conducted among 215 AYA patients with cancer aged 14 to 39 years. A total of 141 patients completed a self-report measure of social functioning within the first 4 months of diagnosis and again at 12 months and 24 months later. RESULTS: AYA patients with cancer were found to have significantly worse social functioning scores around the time of diagnosis (52.0 vs 85.1; P<.001), at the 12-month follow-up (73.1 vs 85.1; P<.001), and at the 24-month follow-up (69.2 vs 85.1; P<.001) when compared with population norms. Significant improvements in social functioning from baseline to the 12-month follow-up were observed; however, social functioning levels remained stable thereafter. Among participants, 9% demonstrated consistently high/normal social functioning, 47% demonstrated improved social functioning, 13% were found to have worsening social functioning, and 32% demonstrated consistently low social functioning. AYA patients with cancer who had consistently low social functioning were more often off treatment at the time of follow-up, reported more physical symptoms and higher levels of distress at baseline and follow-up, and perceived less social support at baseline compared with the other 3 groups. CONCLUSIONS: Although improved over time, social functioning still was found to be compromised 24 months after the primary diagnosis. Nearly one-third of these patients remain at risk of poor social functioning. Reducing physical symptoms and psychological distress and enhancing social support by interventions during the period after treatment may potentially help these young survivors to better reintegrate into society. Cancer 2017;123:2743-51. © 2017 American Cancer Society.
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Nível de Saúde , Neoplasias/psicologia , Qualidade de Vida/psicologia , Comportamento Social , Adolescente , Adulto , Feminino , Humanos , Estudos Longitudinais , Masculino , Estudos Prospectivos , Risco , Apoio Social , Adulto JovemRESUMO
OBJECTIVE: To assess clinical and nonclinical characteristics associated with the use of pediatric inpatient rehabilitation services among children with traumatic injuries. We hypothesized there would be no nonclinical variations in the use of pediatric inpatient rehabilitation services. STUDY DESIGN: Retrospective analysis of 1139 patients who were injured seriously (0-18 years of age) from our institutional trauma registry (2004-2014). Patients' nonclinical and clinical characteristics were analyzed. We used a full matching technique to compare characteristics between those admitted to rehabilitation (cases) to those discharged home (controls). We matched patients by age category, sex, maximum Abbreviated Injury Scale, and body region of maximum Abbreviated Injury Scale. We used survey-based multivariate logistic regression to identify characteristics associated with inpatient rehabilitation services, controlling for multiple injuries, distance from home to rehabilitation center, year of service, hospital length of stay, and clinically relevant interactions. RESULTS: Ninety-eight patients (8.6%) were admitted to inpatient rehabilitation and 968 (85.0%) were discharged home. Black and other minority patients had increased odds of receiving inpatient rehabilitation compared with white patients (OR, 7.6 [P< .001] and OR, 1.6 [P= .03], respectively). Patients with private compared with public insurance had increased odds of receiving inpatient rehabilitation (OR, 2.4; P< .001). CONCLUSIONS: Pediatric inpatient rehabilitation beds are a scarce resource that should be available to those with the greatest clinical need. The mechanism creating differences in the use of inpatient rehabilitation based on nonclinical characteristics such as race/ethnicity or insurance status must be understood to prevent disparities in access to inpatient rehabilitation services.
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Pacientes Internados/estatística & dados numéricos , Ferimentos e Lesões/diagnóstico , Ferimentos e Lesões/reabilitação , Escala Resumida de Ferimentos , Adolescente , Fatores Etários , California , Estudos de Casos e Controles , Criança , Pré-Escolar , Estudos de Coortes , Feminino , Humanos , Escala de Gravidade do Ferimento , Tempo de Internação , Modelos Logísticos , Masculino , Análise Multivariada , Seleção de Pacientes , Prognóstico , Sistema de Registros , Estudos Retrospectivos , Medição de Risco , Fatores Sexuais , Taxa de Sobrevida , Centros de Traumatologia , Resultado do Tratamento , Ferimentos e Lesões/mortalidadeRESUMO
OBJECTIVE: Theories of posttraumatic growth suggest that some degree of distress is necessary to stimulate growth; yet, investigations of the relationship between stress and growth following trauma are mixed. This study aims to understand the relationship between posttraumatic stress symptoms and posttraumatic growth in adolescent and young adult (AYA) cancer patients. METHOD: 165 AYA patients aged 14-39 years at diagnosis completed standardized measures of posttraumatic stress and posttraumatic growth at 12 months following diagnosis. Locally weighted scatterplot smoothing and regression were used to examine linear and curvilinear relationships between posttraumatic stress and posttraumatic growth. RESULTS: No significant relationships between overall posttraumatic stress severity and posttraumatic growth were observed at 12-month follow-up. However, curvilinear relationships between re-experiencing (a posttraumatic stress symptom) and two of five posttraumatic growth indicators (New Possibilities, Personal Strengths) were observed. CONCLUSION: Findings suggest that re-experiencing is associated with some aspects of posttraumatic growth but not others. Although re-experiencing is considered a symptom of posttraumatic stress disorder, it also may represent a cognitive process necessary to achieve personal growth for AYAs. Findings call into question the supposed psychopathological nature of re-experiencing and suggest that re-experiencing, as a cognitive process, may be psychologically adaptive. Opportunities to engage family, friends, cancer survivors, or health care professionals in frank discussions about fears, worries, or concerns may help AYAs re-experience cancer in a way that enhances their understanding of what happened to them and contributes to positive adaptation to life after cancer.
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Adaptação Psicológica , Neoplasias/psicologia , Transtornos de Estresse Pós-Traumáticos/psicologia , Adolescente , Adulto , Feminino , Humanos , Masculino , Análise de Regressão , Índice de Gravidade de Doença , Estresse Psicológico/psicologia , Adulto JovemRESUMO
PURPOSE: Identifying at-risk adolescent and young adult (AYA) cancer patients and referring them to age-appropriate psychosocial support services may be instrumental in reducing psychological distress and promoting psychosocial adaptation. The purpose of this study is to identify trajectories of clinically significant levels of distress throughout the first year following diagnosis and to distinguish factors, including supportive care service use, that predict the extent to which AYAs report distress. METHODS: In this prospective multisite study, 215 AYAs aged 15-39 years were assessed for psychological distress and psychosocial support service use within the first 4 months of diagnosis and again 6 and 12 months later. On the basis of distress scores, respondents were assigned to one of four distress trajectory groups (Resilient, Recovery, Delayed, and Chronic). Multiple logistic regression analyses examined whether demographics, clinical variables, and reports of unsatisfied need for psychosocial support were associated with distress trajectories over 1 year. RESULTS: Twelve percent of AYAs reported clinically significant chronic distress throughout the first 12 months following diagnosis. An additional 15% reported delayed distress. Substantial proportions of AYAs reported that needs for information (57%), counseling (41%), and practical support (39%) remained unsatisfied at 12 months following diagnosis. Not getting counseling needs met, particularly with regard to professional mental health services, was observed to be significantly associated with distress over time. CONCLUSIONS: Substantial proportions of AYAs are not utilizing psychosocial support services. Findings suggest the importance of identifying psychologically distressed AYAs and addressing their needs for mental health counseling throughout a continuum of care.
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Ansiedade/psicologia , Depressão/psicologia , Avaliação das Necessidades , Neoplasias/psicologia , Resiliência Psicológica , Apoio Social , Estresse Psicológico/psicologia , Adaptação Psicológica , Adolescente , Adulto , Ansiedade/terapia , Estudos de Coortes , Depressão/terapia , Feminino , Humanos , Modelos Logísticos , Estudos Longitudinais , Masculino , Serviços de Saúde Mental/estatística & dados numéricos , Análise Multivariada , Estudos Prospectivos , Estresse Psicológico/terapia , Adulto JovemRESUMO
[This corrects the article DOI: 10.1371/journal.pone.0278354.].
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BACKGROUND: Adolescents and young adults (AYAs) with cancer demonstrate biomedical risks and psychosocial issues distinct from those of children or older adults. In this study, the authors examined and compared the extent to which AYAs treated in pediatric or adult oncology settings reported use of, and unmet need for, psychosocial support services. METHODS: Within 4 months of initial cancer diagnosis, 215 AYAs ages 14 to 39 years (99 from pediatric care settings and 116 from adult care settings; 75% response rate) were assessed for reporting use of information resources, emotional support services, and practical support services. Statistical analyses derived odds ratios and 95% confidence intervals for service use and unmet needs after controlling for race, employment/school status, sex, relationship status, severity of cancer, treatment, and treatment-related side effects. RESULTS: AYAs ages 20 to 29 years were significantly less likely than teens and older patients ages 30 to 39 years to report using professional mental health services and were significantly more likely to report an unmet need with regard to cancer information, infertility information, and diet/nutrition information. Compared with teens who were treated in pediatric facilities, AYAs who were treated in adult facilities were more likely to report an unmet need for age-appropriate Internet sites, professional mental health services, camp/retreats programs, transportation assistance, and complementary and alternative health services. CONCLUSIONS: Substantial proportions of AYAs are not getting their psychosocial care needs met. Bolstering psychosocial support staff and patient referral to community-based social service agencies and reputable Internet resources may enhance care and improve quality of life for AYAs.
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Serviços de Saúde do Adolescente , Neoplasias/psicologia , Apoio Social , Adolescente , Adulto , Feminino , Humanos , Masculino , Serviços de Saúde Mental/estatística & dados numéricos , Avaliação das Necessidades , Neoplasias/terapia , Educação de Pacientes como Assunto , Estudos Prospectivos , Adulto JovemRESUMO
OBJECTIVES: Post-traumatic stress symptoms (PTSS) have been identified as a meaningful indicator of distress in cancer survivors. Distinct from young adult survivors of childhood cancer, young people diagnosed with cancer as adolescents and young adults (AYAs) face unique psychosocial issues; however, there is little published research of PTSS in the AYA population. This study examines prevalence and predictors of PTSS among AYAs with cancer. METHODS: As part of a longitudinal study of AYAs with cancer, 151 patients aged 15-39 years completed mailed surveys at 6 and 12 months post-diagnosis. Severity of PTSS was estimated at 6 and 12 months post-diagnosis. Multiple regression analyses were conducted to investigate the predictive effects of socio-demographic and clinical characteristics on changes in PTSS over time. RESULTS: At 6 and 12 months, respectively, 39% and 44% of participants reported moderate to severe levels of PTSS; 29% had PTSS levels suggestive of post-traumatic stress disorder. No significant differences in severity of PTSS between 6 and 12 months were observed. Regression analyses suggested that a greater number of side effects were associated with higher levels of PTSS at 6 months. Currently receiving treatment, having surgical treatment, diagnosis of a cancer type with a 90-100% survival rate, remaining unemployed/not in school, and greater PTSS at 6 months were associated with higher levels of PTSS at 12 months. CONCLUSIONS: Post-traumatic stress symptoms were observed as early as 6 months following diagnosis and remained stable at 12-month follow-up. The development of early interventions for reducing distress among AYA patients in treatment is recommended.
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Neoplasias/psicologia , Transtornos de Estresse Pós-Traumáticos/epidemiologia , Sobreviventes/psicologia , Adaptação Psicológica , Adolescente , Adulto , Feminino , Seguimentos , Nível de Saúde , Humanos , Masculino , Neoplasias/complicações , Neoplasias/epidemiologia , Prevalência , Estudos Prospectivos , Qualidade de Vida , Análise de Regressão , Fatores de Risco , Índice de Gravidade de Doença , Fatores Socioeconômicos , Transtornos de Estresse Pós-Traumáticos/diagnóstico , Transtornos de Estresse Pós-Traumáticos/etiologia , Transtornos de Estresse Pós-Traumáticos/psicologia , Adulto JovemRESUMO
This study examines childhood cancer survival rates and prognostic factors related to survival in the majority Hispanic population of South Texas. The population-based cohort study used Texas Cancer Registry data (1995-2017) to examine survival and prognostic factors. Cox proportional hazard models and Kaplan-Meier survival curves were used for survival analyses. The 5-year relative survival rate for 7,999 South Texas cancer patients diagnosed at 0-19 years was 80.3% for all races/ethnicities. Hispanic patients had statistically significant lower 5-year relative survival rates than non-Hispanic White (NHW) patients for male and female together diagnosed at age≥5 years. When comparing survival among Hispanic and NHW patients for the most common cancer, acute lymphocytic leukemia (ALL), the difference was most significant in the 15-19 years age range, with 47.7% Hispanic patients surviving at 5 years compared to 78.4% of NHW counterparts. The multivariable-adjusted analysis showed that males had statistically significant 13% increased mortality risk than females [hazard ratio (HR): 1.13, 95% confidence interval (CI):1.01-1.26] for all cancer types. Comparing to patients diagnosed at ages 1-4 years, patients diagnosed at age < 1 year (HR: 1.69, 95% CI: 1.36-2.09), at 10-14 year (HR: 1.42, 95% CI: 1.20-1.68), or at 15-19 years (HR: 1.40, 95% CI: 1.20-1.64) had significant increased mortality risk. Comparing to NHW patients, Hispanic patients showed 38% significantly increased mortality risk for all cancer types, 66% for ALL, and 52% for brain cancer. South Texas Hispanic patients had lower 5-year relative survival than NHW patients especially for ALL. Male gender, diagnosis at age<1 year or 10-19 years were also associated with decreased childhood cancer survival. Despite advances in treatment, Hispanic patients lag significantly behind NHW patients. Further cohort studies in South Texas are warranted to identify additional factors affecting survival and to develop interventional strategies.
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Neoplasias , Populações Vulneráveis , Humanos , Masculino , Criança , Feminino , Pessoa de Meia-Idade , Pré-Escolar , Lactente , Estudos de Coortes , Texas/epidemiologia , Neoplasias/epidemiologia , BrancosRESUMO
PURPOSE: Patients with cirrhosis needing anticoagulation therapy have historically been prescribed warfarin. New retrospective research has concluded that in patients with cirrhosis direct oral anticoagulants (DOACs) have similar or lower bleeding rates compared with that of warfarin. This study compares the safety and efficacy of DOACs with that of warfarin in patients with cirrhosis. METHODS: A retrospective chart review was conducted in adult patients with cirrhosis taking either apixaban, dabigatran, edoxaban, rivaroxaban, or warfarin. Exclusion criteria consisted of patients prescribed triple antithrombotic therapy (dual antiplatelet therapy plus an anticoagulant) and indications other than nonvalvular atrial fibrillation (NVAF) and venous thromboembolism (VTE). The primary endpoint was all-cause bleeding, and the secondary endpoints were failed efficacy and major bleeding as defined by the International Society on Thrombosis and Haemostasis in 2005. Failed efficacy was a combination endpoint including the development of VTE, stroke, myocardial infarction and/or death. Patient data were collected from the Computerized Patient Record System from October 31, 2014 to October 31, 2018. RESULTS: The study included 42 patients in the DOAC group and 37 patients in the warfarin group. Baseline characteristics were not significantly different between groups except for the Child-Turcotte-Pugh score, Model for End-Stage Liver Disease score, international normalized ratio, and number of days on anticoagulation therapy. The rate of all-cause bleeding in the DOAC group was 16.7% (n = 7) vs 21.6% (n = 8) in the warfarin group (P = .7). The rate of major bleeding in the DOAC group was 2.4% (n = 1) vs 5.4% (n = 2) in the warfarin group (P = .6). The rate of failed efficacy in the DOAC group was 7.1% (n = 3) compared with 8.1% (n = 3) in the warfarin group (P = .9). Subgroup analysis of allcause bleeding did not identify any significant trends between groups. CONCLUSIONS: There were no statistically significant differences identified between the rates of all-cause bleeding, major bleeding, and failed efficacy between the DOACs and warfarin groups. DOACs may be a safe alternative to warfarin in patients with cirrhosis requiring anticoagulation for NVAF or VTE, but large randomized trials are required to confirm these results.
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OBJECTIVE: This study investigated profiles of emotional distress and growth in adolescents and young adults (AYAs) with cancer. Clinical, demographic, and psychosocial factors were examined for their potential to distinguish these profiles and predict health-related quality of life (HRQoL) of AYAs with cancer. METHOD: This was a multicenter, longitudinal study of AYAs diagnosed with cancer at 14-39 years of age. Participants were assessed 3 times over 24 months following a baseline survey administered at diagnosis. Four profiles (resilient, resilient growth, distressed, distressed growth) were derived using published cutoff points on standardized measures of depression, anxiety, posttraumatic stress disorder, and posttraumatic growth. Mixed-effects models were used to examine profile correlates and the extent to which profiles were associated with HRQoL. RESULTS: Among 179 participants at Time 1, the proportion of profiles ranged from 18.8% for the resilient profile to 30.4% for the distressed-growth profile. These proportions remained consistent over time. Factors that appeared to distinguish these profiles included work or school status, sex, race, age at diagnosis, treatment status, prognosis, and personality characteristics. When compared to AYAs with resilient-growth profiles, HRQoL was significantly worse for AYAs reporting distressed and distressed-growth profiles, controlling for demographic, clinical, and social characteristics. CONCLUSION: The current study found 4 patterns of psychological adjustment in AYAs with cancer. The resilient-growth profile was associated with better HRQoL, whereas distressed and distressed-growth profiles were associated with worse HRQoL. (PsycInfo Database Record (c) 2020 APA, all rights reserved).
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Neoplasias/psicologia , Angústia Psicológica , Qualidade de Vida/psicologia , Adolescente , Adulto , Feminino , Humanos , Estudos Longitudinais , Masculino , Adulto JovemRESUMO
Bridging with enoxaparin rather than heparin has the potential to reduce the length of hospital stay, incidence of nosocomial infections, and cost of hospitalization.
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BACKGROUND: Pantothenate kinase-associated neurodegeneration (PKAN) is a rare genetic disorder characterised by progressive generalised dystonia and brain iron accumulation. We assessed whether the iron chelator deferiprone can reduce brain iron and slow disease progression. METHODS: We did an 18-month, randomised, double-blind, placebo-controlled trial (TIRCON2012V1), followed by a pre-planned 18-month, open-label extension study, in patients with PKAN in four hospitals in Germany, Italy, England, and the USA. Patients aged 4 years or older with a genetically confirmed diagnosis of PKAN, a total score of at least 3 points on the Barry-Albright Dystonia (BAD) scale, and no evidence of iron deficiency, neutropenia, or abnormal hepatic or renal function, were randomly allocated (2:1) to receive an oral solution of either deferiprone (30 mg/kg per day divided into two equal doses) or placebo for 18 months. Randomisation was done with a centralised computer random number generator and with stratification based on age group at onset of symptoms. Patients were allocated to groups by a randomisation team not masked for study intervention that was independent of the study. Patients, caregivers, and investigators were masked to treatment allocation. Co-primary endpoints were the change from baseline to month 18 in the total score on the BAD scale (which measures severity of dystonia in eight body regions) and the score at month 18 on the Patient Global Impression of Improvement (PGI-I) scale, which is a patient-reported interpretation of symptom improvement. Efficacy analyses were done on all patients who received at least one dose of the study drug and who provided a baseline and at least one post-baseline efficacy assessment. Safety analyses were done for all patients who received at least one dose of the study drug. Patients who completed the randomised trial were eligible to enrol in a single-arm, open-label extension study of another 18 months, in which all participants received deferiprone with the same regimen as the main study. The trial was registered on ClinicalTrials.gov, number NCT01741532, and EudraCT, number 2012-000845-11. FINDINGS: Following a screening of 100 prospective patients, 88 were randomly assigned to the deferiprone group (n=58) or placebo group (n=30) between Dec 13, 2012, and April 21, 2015. Of these, 76 patients completed the study (49 in the deferiprone group and 27 in the placebo group). After 18 months, the BAD score worsened by a mean of 2·48 points (SE 0·63) in patients in the deferiprone group versus 3·99 points (0·82) for patients in the control group (difference -1·51 points, 95% CI -3·19 to 0·16, p=0·076). No subjective change was detected as assessed by the PGI-I scale: mean scores at month 18 were 4·6 points (SE 0·3) for patients in the deferiprone group versus 4·7 points (0·4) for those in the placebo group (p=0·728). In the extension study, patients continuing deferiprone retained a similar rate of disease progression as assessed by the BAD scale (1·9 points [0·5] in the first 18 months vs 1·4 points [0·4] in the second 18 months, p=0·268), whereas progression in patients switching from placebo to deferiprone seemed to slow (4·4 points [1·1] vs 1·4 points [0·9], p=0·021). Patients did not detect a change in their condition after the additional 18 months of treatment as assessed by the PGI-I scale, with mean scores of 4·1 points [0·2] in the deferiprone-deferiprone group and of 4·7 points [0·3] in the placebo-deferiprone group. Deferiprone was well tolerated and adverse events were similar between the treatment groups, except for anaemia, which was seen in 12 (21%) of 58 patients in the deferiprone group, but was not seen in any patients in the placebo group. No patient discontinued therapy because of anaemia, and three discontinued because of moderate neutropenia. There was one death in each group of the extension study and both were secondary to aspiration. Neither of these events was considered related to deferiprone use. INTERPRETATION: Deferiprone was well tolerated, achieved target engagement (lowering of iron in the basal ganglia), and seemed to somewhat slow disease progression at 18 months, although not significantly, as assessed by the BAD scale. These findings were corroborated by the results of an additional 18 months of treatment in the extension study. The subjective PGI-I scale was largely unchanged during both study periods, indicating that might not be an adequate tool for assessment of disease progression in patients with PKAN. Our trial provides the first indication of a decrease in disease progression in patients with neurodegeneration with brain iron accumulation. The extensive information collected and long follow-up of patients in the trial will improve the definition of appropriate endpoints, increase the understanding of the natural history, and thus help to shape the design of future trials in this ultra-orphan disease. FUNDING: European Commission, US Food and Drug Administration, and ApoPharma Inc.
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Deferiprona/uso terapêutico , Quelantes de Ferro/uso terapêutico , Neurodegeneração Associada a Pantotenato-Quinase/tratamento farmacológico , Adolescente , Adulto , Criança , Pré-Escolar , Deferiprona/efeitos adversos , Progressão da Doença , Método Duplo-Cego , Feminino , Humanos , Quelantes de Ferro/efeitos adversos , Masculino , Pessoa de Meia-Idade , Resultado do Tratamento , Adulto JovemRESUMO
Personality may affect the way adolescents and young adults (AYAs) with cancer report health-related quality of life (HRQoL). Patients aged 15-39 years (n = 165) completed a survey at 12-16 months postdiagnosis. The survey included questions on HRQoL (SF-36), distress Brief Symptom Inventory-18, and personality (NEO-Five-Factor Inventory). Personality traits were not associated with physical HRQoL. The personality trait neuroticism was negatively associated with mental HRQoL (ß = -0.37; p < 0.001) and positively with psychological distress (ß = 0.47; p < 0.001). Hierarchical regression and mediation analyses indicated that psychological distress fully mediated the association between neuroticism and mental HRQoL. Findings emphasize the importance of psychosocial intervention for distress in AYAs with cancer.
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Ansiedade/psicologia , Nível de Saúde , Neoplasias/psicologia , Personalidade , Qualidade de Vida/psicologia , Estresse Psicológico/psicologia , Adolescente , Adulto , Feminino , Humanos , Modelos Lineares , Masculino , Neoplasias/terapia , Adulto JovemRESUMO
Purpose To examine changes in health-related quality of life (HRQoL) and its predictors during the first 2 years after initial cancer diagnosis in adolescent and young adult (AYA) patients with cancer. Patients and Methods A multicenter, longitudinal, prospective study was conducted among a diverse sample of AYA patients with cancer ages 15 to 39 years. One hundred seventy-six patients (75% response) completed a self-report measure of HRQoL (Short Form-36 [SF-36]) within the first 4 months after diagnosis and again 12 and 24 months later. Linear mixed models with random intercepts and slopes estimated changes in QoL. Results Recently diagnosed AYA patients with cancer had significantly worse physical component scale (PCS) scores (38.7 v 52.8; P < .001) and mental component scale (MCS) scores (42.9 v 48.9; P < .001) when compared with population norms. Significant improvements in PCS and MCS scores from baseline to 24-month follow-up were observed; however, these increases were largest during the first 12 months. At the 24-month follow-up, AYA patients still had significantly lower PCS scores (48.0 v 52.8; P < .001) and MCS scores (45.8 v 48.9; P = .002) when compared with population norms. Multivariable analyses revealed that improvements in PCS and MCS scores were primarily a function of being off-treatment and being involved in school or work. PCS but not MCS scores were worse for AYA patients diagnosed with cancers with poorer prognoses. Conclusion Although HRQoL improved over time, it was still compromised 24 months after primary diagnosis. Given relatively little observed improvement in HRQoL during the 12- to 24-month period after diagnosis, AYA patients may benefit from supportive care interventions administered during the second year after diagnosis.
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Neoplasias/fisiopatologia , Adolescente , Adulto , Fatores Etários , Feminino , Humanos , Estudos Longitudinais , Masculino , Neoplasias/psicologia , Neoplasias/terapia , Estudos Prospectivos , Qualidade de Vida , Autorrelato , Adulto JovemRESUMO
BACKGROUND: Osteonecrosis of the femoral head is a common complication in patients with sickle cell disease, and collapse of the femoral head occurs in 90% of patients within five years after the diagnosis of the osteonecrosis. However, the efficacy of hip core decompression to prevent the progression of osteonecrosis in these patients is still controversial. METHODS: In a prospective multicenter study, we evaluated the safety of hip core decompression and compared the results of decompression and physical therapy with those of physical therapy alone for the treatment of osteonecrosis of the femoral head in patients with sickle cell disease. Forty-six patients (forty-six hips) with sickle cell disease and Steinberg Stage-I, II, or III osteonecrosis of the femoral head were randomized to one of two treatment arms: (1) hip core decompression followed by a physical therapy program or (2) a physical therapy program alone. Eight patients withdrew from the study, leaving thirty-eight who participated. RESULTS: Seventeen patients (seventeen hips) underwent decompression combined with physical therapy, and no intraoperative or immediate postoperative complications occurred. Twenty-one patients (twenty-one hips) were treated with physical therapy alone. After a mean of three years, the hip survival rate was 82% in the group treated with decompression and physical therapy and 86% in the group treated with physical therapy alone. According to a modification of the Harris hip score, the mean clinical improvement was 18.1 points for the patients treated with hip core decompression and physical therapy compared with 15.7 points for those treated with physical therapy alone. With the numbers studied, the differences were not significant. CONCLUSIONS: In this randomized prospective study, physical therapy alone appeared to be as effective as hip core decompression followed by physical therapy in improving hip function and postponing the need for additional surgical intervention at a mean of three years after treatment.
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Anemia Falciforme/epidemiologia , Descompressão Cirúrgica , Necrose da Cabeça do Fêmur/epidemiologia , Necrose da Cabeça do Fêmur/terapia , Colo do Fêmur/cirurgia , Modalidades de Fisioterapia , Adulto , Artroplastia de Quadril , Terapia Combinada , Comorbidade , Feminino , Necrose da Cabeça do Fêmur/cirurgia , Seguimentos , Humanos , Masculino , Prevalência , Estudos Prospectivos , Resultado do Tratamento , Suporte de CargaRESUMO
Bone and joint disorders are the most common cause of chronic pain in patients who have sickle cell disease. The femoral head is the most common area of bone destruction in sickle cell patients, although other disease-related problems include avascular necrosis of the humeral head, changes in the thoracic and lumbar spine, infection with encapsulated organisms (Salmonella and Staphylococcus aureus are the most common), bone marrow disturbances, and dental effects. Complications can occur at any location: epiphyseal, metaphyseal, or diaphyseal. The location and the extensiveness of the problems determine the pain and structural damage. The hip joint is particularly vulnerable in sickle cell disease. This article highlights aspects of sickle cell disease that affect healthy bone and joint function and discusses treatment options.
Assuntos
Anemia Falciforme , Doenças Ósseas , Artropatias , Anemia Falciforme/complicações , Anemia Falciforme/fisiopatologia , Anemia Falciforme/terapia , Doenças Ósseas/complicações , Doenças Ósseas/fisiopatologia , Doenças Ósseas/terapia , Humanos , Artropatias/complicações , Artropatias/fisiopatologia , Artropatias/terapia , Fatores de RiscoRESUMO
OBJECTIVE: A quality improvement tool is provided to improve pharmacy workflow with the goal of minimizing errors caused by workflow issues. This study involved workflow evaluation and reorganization, and staff opinions of these proposed changes. PRACTICE DESCRIPTION: The study pharmacy was an outpatient pharmacy in the Tucson area. However, the quality improvement tool may be applied in all pharmacy settings, including but not limited to community, hospital, and independent pharmacies. PRACTICE INNOVATION: This tool can help the user to identify potential workflow problem spots, such as high-traffic areas through the creation of current and proposed workflow diagrams. Creating a visual representation can help the user to identify problem spots and to propose changes to optimize workflow. It may also be helpful to assess employees' opinions of these changes. CONCLUSION: The workflow improvement tool can be used to assess where improvements are needed in a pharmacy's floor plan and workflow. Suggestions for improvements in the study pharmacy included increasing the number of verification points and decreasing high traffic areas in the workflow. The employees of the study pharmacy felt that the proposed changes displayed greater continuity, sufficiency, accessibility, and space within the pharmacy.
Assuntos
Assistência Ambulatorial/organização & administração , Erros de Medicação/prevenção & controle , Serviço de Farmácia Hospitalar/organização & administração , Fluxo de Trabalho , Assistência Ambulatorial/normas , Arquitetura de Instituições de Saúde , Humanos , Farmácias/organização & administração , Farmácias/normas , Serviço de Farmácia Hospitalar/normas , Projetos Piloto , Melhoria de QualidadeRESUMO
PURPOSE: To examine prevalence and changes in symptoms of psychological distress over 1 year after initial cancer diagnosis in adolescent and young adult (AYA) patients with cancer. Sociodemographic and clinical predictors of changes in distress were examined. PATIENTS AND METHODS: In this multisite, longitudinal, prospective study of an ethnically diverse sample, 215 patients age 14 to 39 years were assessed for psychological distress within the first 4 months of diagnosis and again 6 and 12 months later. Linear mixed models with random intercept and slope estimated changes in distress, as measured by the Brief Symptom Inventory-18 (BSI-18). RESULTS: Within the first 4 months of diagnosis, 60 respondents (28%) had BSI-18 scores suggesting caseness for distress. On average, distress symptoms exceeded population norms at the time of diagnosis, dipped at the 6-month follow-up, but increased to a level exceeding population norms at the 12-month follow-up. A statistically significant decline in distress over 1 year was observed; however, the gradient of change was not clinically significant. Multivariate analyses revealed that the reduction in distress over time was primarily a function of being off treatment and involved in school or work. Notably, cancer type or severity was not associated with distress. CONCLUSION: Findings emphasize the importance of early psychosocial intervention for distress in AYAs as well as the need to manage treatment-related symptoms and facilitate AYAs' involvement in work or school to the extent possible. Continued research is needed to understand how distress relates to quality of life, functional outcomes, treatment, and symptom burden throughout the continuum of care.