RESUMO
Rationale: Pulmonary complications contribute significantly to nonrelapse mortality following hematopoietic stem cell transplantation (HCT). Identifying patients at high risk can help enroll such patients into clinical studies to better understand, prevent, and treat posttransplantation respiratory failure syndromes. Objectives: To develop and validate a prediction model to identify those at increased risk of acute respiratory failure after HCT. Methods: Patients underwent HCT between January 1, 2019, and December 31, 2021, at one of three institutions. Those treated in Rochester, MN, formed the derivation cohort, and those treated in Scottsdale, AZ, or Jacksonville, FL, formed the validation cohort. The primary outcome was the development of acute respiratory distress syndrome (ARDS), with secondary outcomes including the need for invasive mechanical ventilation (IMV) and/or noninvasive ventilation (NIV). Predictors were based on prior case-control studies. Measurements and Main Results: Of 2,450 patients undergoing stem cell transplantation, there were 1,718 hospitalizations (888 patients) in the training cohort and 1,005 hospitalizations (470 patients) in the test cohort. A 22-point model was developed, with 11 points from prehospital predictors and 11 points from posttransplantation or early (<24-h) in-hospital predictors. The model performed well in predicting ARDS (C-statistic, 0.905; 95% confidence interval [CI], 0.870-0.941) and the need for IMV and/or NIV (C-statistic, 0.863; 95% CI, 0.828-0.898). The test cohort differed markedly in demographic, medical, and hematologic characteristics. The model also performed well in this setting in predicting ARDS (C-statistic, 0.841; 95% CI, 0.782-0.900) and the need for IMV and/or NIV (C-statistic, 0.872; 95% CI, 0.831-0.914). Conclusions: A novel prediction model incorporating data elements from the pretransplantation, posttransplantation, and early in-hospital domains can reliably predict the development of post-HCT acute respiratory failure.
Assuntos
Lesão Pulmonar , Síndrome do Desconforto Respiratório , Insuficiência Respiratória , Humanos , Transplante de Medula Óssea/efeitos adversos , Lesão Pulmonar/complicações , Estudos de Coortes , Síndrome do Desconforto Respiratório/terapia , Síndrome do Desconforto Respiratório/complicações , Insuficiência Respiratória/terapiaRESUMO
BACKGROUND: This study examines patients' understanding of health insurance terms and concepts and quantifies health insurance literacy (HIL) levels by key sociodemographic factors. METHODS: This study included 393 adult patients with cancer (>18 years old) receiving treatment in two ambulatory infusion centers: Mayo Clinic in Phoenix, Arizona and the University of Mississippi Medical Center in Jackson, Mississippi. Respondents' perceptions of their HIL were assessed using the Health Insurance Literacy Measure (HILM), a validated 21-item measure of a consumer's ability to select and use health insurance (HIL self-efficacy). Respondents' knowledge of health insurance concepts (HIL knowledge) was measured using 10 items created by the Kaiser Family Foundation. The number of correct answers was categorized into three levels: 0-4 (low knowledge), 5-6 (moderate knowledge), and 7-10 (high knowledge). Multivariable logistic regressions were used to compare correct answers to HIL knowledge questions by HIL self-efficacy. RESULTS: Nearly three-quarters of patients had high HIL self-efficacy and high HIL knowledge (70.5%), understanding basic insurance terms, such as premiums and deductibles. Relatively low percentages of patients correctly answered questions about the meaning of provider networks, health insurance formularies, and calculating out-of-pocket spending in scenarios when insurers pay a portion of allowed charges. Lower HIL knowledge was more common among patients with less educational attainment (Assuntos
Letramento em Saúde
, Seguro Saúde
, Neoplasias
, Humanos
, Feminino
, Masculino
, Pessoa de Meia-Idade
, Neoplasias/terapia
, Adulto
, Idoso
, Pacientes Ambulatoriais/estatística & dados numéricos
, Autoeficácia
, Cobertura do Seguro
RESUMO
Among 210 patients with myelodysplastic syndromes (MDSs) with del(5q), molecular information was available at diagnosis or at least 3 months before leukaemic transformation in 146 cases. Multivariate analysis identified therapy-related setting (p = 0.02; HR 2.3) and TP53 variant allele frequency (VAF) ≥22% (p < 0.01; HR 2.8), but not SF3B1 mutation (p = 0.65), as independent risk factors for survival. Median survival was 11.7 versus 4 years (5/10-year survival 73%/52% vs. 42%/14%) in the absence (N = 112) versus presence (N = 34) of ≥1 risk factors; leukaemia-free survival was affected by TP53 VAF ≥22% (p < 0.01). Such information might inform treatment decision-making in MDS-del(5q) regarding allogeneic stem cell transplant.
Assuntos
Síndromes Mielodisplásicas , Humanos , Síndromes Mielodisplásicas/genética , Síndromes Mielodisplásicas/terapia , Síndromes Mielodisplásicas/diagnóstico , Frequência do Gene , Mutação , Prognóstico , Deleção Cromossômica , Cromossomos Humanos Par 5/genética , Proteína Supressora de Tumor p53/genéticaRESUMO
Cytologic abnormalities of atypical mast cells in mastocytosis. The mature mast cells have oval-shaped nuclei, cytoplasmic hypogranulation and spindle-shaped cytology. or well-differentiated displaying a round nucleus with condensed chromatin, and abundant dense cytoplasmic granulations. Immature mast cells include promastocytes and metachromatic blast-like forms.
Assuntos
Leucemia de Mastócitos , Mastocitose , Humanos , MastócitosRESUMO
Venetoclax + hypomethylating agent (Ven-HMA) is currently the standard frontline therapy for older/unfit patients with newly diagnosed acute myeloid leukemia (ND-AML). Our objective in the current retrospective study of 301 adult patients (median age 73 years; 62% de novo) with ND-AML was to identify molecular predictors of treatment response to Ven-HMA and survival; European LeukemiaNet (ELN) genetic risk assignment was favorable 15%, intermediate 16%, and adverse 69%. Complete remission, with (CR) or without (CRi), count recovery, was documented in 182 (60%) patients. In multivariable analysis, inclusive of mutations only, "favorable" predictors of CR/CRi were NPM1 (86% vs. 56%), IDH2 (80% vs. 58%), and DDX41 (100% vs. 58%) and "unfavorable" TP53 (40% vs. 67%), FLT3-ITD (36% vs. 63%), and RUNX1 (44% vs. 64%) mutations; significance was sustained for each mutation after adjustment for age, karyotype, and therapy-related qualification. CR/CRi rates ranged from 36%, in the presence of unfavorable and absence of favorable mutation, to 91%, in the presence of favorable and absence of unfavorable mutation. At median follow-up of 8.5 months, 174 deaths and 41 allogeneic stem cell transplants (ASCT) were recorded. In multivariable analysis, risk factors for inferior survival included failure to achieve CR/CRi (HR 3.4, 95% CI 2.5-4.8), adverse karyotype (1.6, 1.1-2.6), TP53 mutation (1.6, 1.0-2.4), and absence of IDH2 mutation (2.2, 1.0-4.7); these risk factors were subsequently applied to construct an HR-weighted risk model that performed better than the ELN genetic risk model (AIC 1661 vs. 1750): low (n = 130; median survival 28.9 months), intermediate (n = 105; median 9.6 months), and high (n = 66; median 3.1 months; p < .001); survival in each risk category was significantly upgraded by ASCT. The current study identifies genotype signatures for predicting response and proposes a 3-tiered, CR/CRi-based, and genetics-enhanced survival model for AML patients receiving upfront therapy with Ven-HMA.
Assuntos
Compostos Bicíclicos Heterocíclicos com Pontes , Leucemia Mieloide Aguda , Sulfonamidas , Adulto , Humanos , Idoso , Intervalo Livre de Doença , Estudos Retrospectivos , Leucemia Mieloide Aguda/tratamento farmacológico , Leucemia Mieloide Aguda/genética , Genótipo , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêuticoRESUMO
Overall survival and response rates of 270 patients with newly diagnosed acute myeloid leukemia receiving venetoclax (Ven) plus hypomethylating agent, stratified by Ven dosing schedule (Cycle 1 Ven 14 vs. 21 vs. 28 days).
Assuntos
Compostos Bicíclicos Heterocíclicos com Pontes , Leucemia Mieloide Aguda , Humanos , Compostos Bicíclicos Heterocíclicos com Pontes/efeitos adversos , Sulfonamidas/efeitos adversos , Leucemia Mieloide Aguda/tratamento farmacológico , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversosRESUMO
Social determinants of health, including poverty, contribute significantly to health outcomes in the United States; however, their impact on pediatric hematopoietic cell transplantation (HCT) outcomes is poorly understood. We aimed to identify the association between neighborhood poverty and HCT outcomes for pediatric allogeneic HCT recipients in the Center for International Blood and Marrow Transplant Research database. We assembled 2 pediatric cohorts undergoing first allogeneic HCT from 2006 to 2015 at age ≤18 years, including 2053 children with malignant disease and 1696 children with nonmalignant disease. Neighborhood poverty exposure was defined a priori per the US Census definition as living in a high-poverty ZIP code (≥20% of persons below 100% federal poverty level) and used as the primary predictor in all analyses. Our primary outcome was overall survival (OS), defined as the time from HCT until death resulting from any cause. Secondary outcomes included relapse and transplantation-related mortality (TRM) in malignant disease, acute and chronic graft-versus-host disease, and infection in the first 100 days post-HCT. Among children undergoing transplantation for nonmalignant disease, neighborhood poverty was not associated with any HCT outcome. Among children undergoing transplantation for malignant disease, neighborhood poverty conferred an increased risk of TRM but was not associated with inferior OS or any other transplantation outcome. Among children with malignant disease, a key secondary finding was that children with Medicaid insurance experienced inferior OS and increased TRM compared with those with private insurance. These data suggest opportunities for future investigation of the effects of household-level poverty exposure on HCT outcomes in pediatric malignant disease to inform care delivery interventions.
Assuntos
Transplante de Células-Tronco Hematopoéticas , Pobreza , Determinantes Sociais da Saúde , Adolescente , Causas de Morte , Criança , Pré-Escolar , Doença Crônica/mortalidade , Doença Crônica/terapia , Bases de Dados Factuais , Feminino , Seguimentos , Doença Enxerto-Hospedeiro/epidemiologia , Doença Enxerto-Hospedeiro/etiologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/economia , Transplante de Células-Tronco Hematopoéticas/mortalidade , Transplante de Células-Tronco Hematopoéticas/estatística & dados numéricos , Humanos , Lactente , Infecções/epidemiologia , Cobertura do Seguro/estatística & dados numéricos , Masculino , Medicaid , Neoplasias/mortalidade , Neoplasias/terapia , Recidiva , Análise de Sobrevida , Transplante Homólogo , Resultado do Tratamento , Estados UnidosRESUMO
BACKGROUND: Physical activity can improve physical health for people living with mild cognitive impairment (MCI) and dementia and may have cognitive benefits. Identifying modifiable social factors inhibiting physical activity among this group is needed. We sought to examine the relationship between reported physical activity levels and social determinants of health (SDOH) in a population of older adults living with MCI or dementia. METHODS: This descriptive study included people with a diagnosis of MCI or dementia followed by Community Internal Medicine at Mayo Clinic (Rochester, Minnesota, United States), aged over 55 years, who had a clinic visit between June 1, 2019 and June 30, 2021 and had completed a SDOH questionnaire. We focused on 8 SDOH domains: education, depression, alcohol use, stress, financial resource strain, social connections, food insecurity, and transportation needs. Data were analyzed based on physical activity level (inactive, insufficiently active, sufficiently active). SDOH domains were compared according to physical activity level using the χ2 test and multinomial logistic regression. RESULTS: A total of 3224 persons with MCI (n = 1371) or dementia (n = 1853) who had completed questions on physical activity were included. Of these, 1936 (60%) were characterized as physically inactive and 837 (26%) insufficiently active. Characteristics associated with an increased likelihood of physical inactivity were older age, female sex, obesity, lower education, dementia diagnosis, screening positive for depression and increased social isolation (p < 0.001). CONCLUSIONS: Physical inactivity is common among people living with MCI and dementia. Physical activity levels may be influenced by many factors, highlighting potential areas for intervention.
Assuntos
Disfunção Cognitiva , Demência , Humanos , Feminino , Estados Unidos/epidemiologia , Idoso , Determinantes Sociais da Saúde , Disfunção Cognitiva/epidemiologia , Exercício Físico , Demência/diagnóstico , Inquéritos e QuestionáriosRESUMO
PURPOSE: Medical financial hardship, encompassing material, behavioral, and psychologic domains, has been shown to impair quality of life during and after cancer therapy. We sought to evaluate the change in financial concerns in breast cancer survivors over time and identify those at risk of worsening financial concerns. METHODS: In Mayo Clinic Breast Disease Registry (MCBDR), a prospective cohort of consenting patients seen at Mayo Clinic Rochester within 1 year of their initial breast cancer diagnosis, consenting participants were asked to complete baseline and annual follow-up surveys that included an item on which respondents were asked to report their financial concerns on a linear analogue scale from 0 ("none") to 10 ("constant concerns"). We compared patient-reported financial concern at baseline to that on each patient's most recent survey, with worsening concerns defined as a 1+-point increase. Logistic regression analysis evaluated for possible predictors of worsening financial concerns. RESULTS: One-thousand nine-hundred fifty-seven participants responded to financial concern questions on the baseline and at least one follow-up survey between 2015 and 2020. Three-hundred fifty-seven (18.2%) reported worsening financial concerns. Only baseline financial situation of "enough to pay the bills, but little spare money to buy extra or special things," was associated with a greater likelihood of worsening financial concerns. CONCLUSIONS: More than one in seven breast cancer survivors develop worsening financial concerns within 5 years of diagnosis, and those with less financial security at baseline appear to be most vulnerable. IMPLICATION FOR CANCER SURVIVORS: Financial concerns may worsen over time for breast cancer survivors, and therefore, oncology providers must continue to assess the financial well-being of survivors over time.
Assuntos
Neoplasias da Mama , Sobreviventes de Câncer , Neoplasias , Humanos , Feminino , Sobreviventes de Câncer/psicologia , Qualidade de Vida , Estresse Financeiro , Sobreviventes , Inquéritos e Questionários , Neoplasias/terapiaRESUMO
Health information technology (HIT) is a widely recognized strategy to encourage cancer patients and caregivers to participate in healthcare delivery in a sustainable and cost-effective way. In the context of autologous hematopoietic cell transplant (HSCT), HIT-enabled tools have the potential to effectively engage, educate, support, and optimize outcomes of patients and caregivers in the outpatient setting. This study sought to leverage human-centered design to develop a high-fidelity prototype of a HIT-enabled psychoeducational tool for HSCT caregivers. Phase 1 focuses on breadth and depth of information gathering through a systematic review and semi-structured interviews to determine optimal tool use. Phase 2 engages in human-centered design synthesis and visualization methods to identify key opportunities for the HIT design. Phase 3 employs human-centered design evaluation, engaging caregivers to respond to low-fidelity concepts and scenarios to help co-design an optimal tool for HSCT. This study outlines a hybrid method of healthcare delivery research and human-centered design to develop technology-enabled support for HSCT caregivers. Herein, we present a design methodology for developing a prototype of HIT-enabled psychoeducational tool which can be leveraged to develop future eHealth innovations to optimize HSCT.
Assuntos
Transplante de Células-Tronco Hematopoéticas , Informática Médica , Cuidadores , Atenção à Saúde , Humanos , TecnologiaRESUMO
OBJECTIVE: Outcomes in multiple myeloma (MM) have significantly improved necessitating focus on survivorship. METHODS: We undertook a web-based survey in collaboration with International Myeloma Foundation (IMF) to explore patient awareness and psycho-physical impacts of MM. The survey was viewed on the IMF website by 1,324 individuals from 32 countries. RESULTS: The survey responses were available from 959 individuals, with 62% who completed the survey. Treating doctors were the most frequent source of MM-related information. Only 56% patients admitted full compliance with treatment. Treatment side effects bothered 86% responders, including >50% admitting to pain, peripheral neuropathy and asthenia. Majority (57%) reported some degree of depression, 82% had discontent with their quality of life and only 35% reported being satisfied with their coping mechanisms. Patients ≥65 years of age reported more peripheral neuropathy (p = 0.007) and difficulty with ability to work (p = 0.015). CONCLUSIONS: We report the prevalence of psychologic, social and physical domains as well as patient-physician relationship dynamics. This knowledge can help improve MM survivorship.Introduction.
Assuntos
Mieloma Múltiplo , Humanos , Internet , Mieloma Múltiplo/terapia , Estudos Prospectivos , Qualidade de Vida , Inquéritos e QuestionáriosRESUMO
Patients undergoing hematopoietic cell transplantation (HCT) are at risk for psychological and social impairment given the rigors and multiple sequelae of treatment. The purpose of this pilot study was to test the feasibility of a digital storytelling (DS) intervention for HCT patients, and to examine limited efficacy of the intervention relative to control arm on psychological distress and perceived social support. Adult HCT patients (n = 40, M age = 59.2 years) were enrolled immediately post-HCT and randomly assigned to either DS intervention or information control (IC). DS participants viewed four 3-min personal, emotionally rich digital stories, and IC condition participants viewed four videos containing information about post-HCT care. Feasibility regarding recruitment, enrollment efforts, and change scores from pre- to post-intervention (Δs) on depression, anxiety, and perceived social support were tracked. Fifty-four (51.4%) of 105 eligible patients consented. Forty (74%) patients completed the intervention. All but one completed the post-intervention assessments demonstrating that HCT patients can be recruited and retained for this intervention. On average, perceived social support increased for the DS group (Δ = 0.06) but decreased for the IC group (Δ = - 0.05). Anxiety and depression improved over time in both conditions. Viewing digital stories with content evoking emotional contexts similar to one's own health challenges may improve perceptions of social support among HCT patients. How DS can improve perceived social support for both short-term and longer-term sustained effects in a longitudinal study is an area ripe for additional investigation.
Assuntos
Transplante de Células-Tronco Hematopoéticas , Adulto , Depressão/prevenção & controle , Depressão/psicologia , Estudos de Viabilidade , Humanos , Estudos Longitudinais , Pessoa de Meia-Idade , Projetos PilotoRESUMO
LAY SUMMARY: People who have advanced myelodysplastic syndromes (MDS) may live longer if they get a bone marrow transplant (BMT) instead of other therapies. However, only 15% of people with MDS actually get BMT. Experts say community physicians and transplant physicians should team up with insurance companies and patient advocacy groups to 1) spread this news about lifesaving advances in BMT, 2) ensure that everyone can afford health care, 3) provide emotional support for patients and families, 4) help patients and families get transportation and housing if they need to travel for transplant, and 5) improve care for people of under-represented racial and ethnic backgrounds.
Assuntos
Transplante de Células-Tronco Hematopoéticas , Síndromes Mielodisplásicas , Medula Óssea , Humanos , Síndromes Mielodisplásicas/terapia , Condicionamento Pré-Transplante , Transplante HomólogoRESUMO
BACKGROUND: The association of community factors and outcomes after hematopoietic cell transplantation (HCT) has not been comprehensively described. Using the County Health Rankings and Roadmaps (CHRR) and the Center for International Blood and Marrow Transplant Research (CIBMTR), this study evaluated the impact of community health status on allogeneic HCT outcomes. METHODS: This study included 18,544 adult allogeneic HCT recipients reported to the CIBMTR by 170 US centers in 2014-2016. Sociodemographic, environmental, and community indicators were derived from the CHRR, an aggregate community risk score was created, and scores were assigned to each patient (patient community risk score [PCS]) and transplant center (center community risk score [CCS]). Higher scores indicated less healthy communities. The impact of PCS and CCS on patient outcomes after allogeneic HCT was studied. RESULTS: The median age was 55 years (range, 18-83 years). The median PCS was -0.21 (range, -1.37 to 2.10; standard deviation [SD], 0.42), and the median CCS was -0.13 (range, -1.04 to 0.96; SD, 0.40). In multivariable analyses, a higher PCS was associated with inferior survival (hazard ratio [HR] per 1 SD increase, 1.04; 99% CI, 1.00-1.08; P = .0089). Among hematologic malignancies, a tendency toward inferior survival was observed with a higher PCS (HR, 1.04; 99% CI, 1.00-1.08; P = .0102); a higher PCS was associated with higher nonrelapse mortality (NRM; HR, 1.08; 99% CI, 1.02-1.15; P = .0004). CCS was not significantly associated with survival, relapse, or NRM. CONCLUSIONS: Patients residing in counties with a worse community health status have inferior survival as a result of an increased risk of NRM after allogeneic HCT. There was no association between the community health status of the transplant center location and allogeneic HCT outcomes.
Assuntos
Planejamento em Saúde Comunitária , Neoplasias Hematológicas/epidemiologia , Transplante de Células-Tronco Hematopoéticas/estatística & dados numéricos , Transplante Homólogo/estatística & dados numéricos , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Feminino , Neoplasias Hematológicas/patologia , Neoplasias Hematológicas/terapia , Humanos , Masculino , Pessoa de Meia-Idade , Recidiva Local de Neoplasia/epidemiologia , Recidiva Local de Neoplasia/terapia , Saúde Pública/estatística & dados numéricos , Fatores de Risco , Resultado do Tratamento , Estados Unidos/epidemiologia , Adulto JovemRESUMO
OPINION STATEMENT: Choice of therapy in mycosis fungoides is based on both patient- and lymphoma-specific factors, such as disease characteristics, comorbidities, symptoms and effect on quality of life, potential associated toxicities of therapy, response and tolerance to prior lines of therapy, and convenience and practicality. Generally, we sequence therapies from least toxic, targeted, nonimmunosuppressive to more toxic, immunosuppressive and from single agent to multiple agents, as necessary. If more toxic, immunosuppressive agents are required to alleviate disease burden or symptoms, we generally use them just long enough to control the disease, then transition to a maintenance regimen with less toxic, less immunosuppressive agents.
Assuntos
Micose Fungoide/terapia , Neoplasias Cutâneas/terapia , Biomarcadores Tumorais , Tomada de Decisão Clínica , Terapia Combinada/efeitos adversos , Terapia Combinada/métodos , Gerenciamento Clínico , Suscetibilidade a Doenças , Humanos , Micose Fungoide/diagnóstico , Micose Fungoide/etiologia , Gradação de Tumores , Estadiamento de Neoplasias , Neoplasias Cutâneas/diagnóstico , Neoplasias Cutâneas/etiologia , Resultado do TratamentoRESUMO
Effective immunosuppressive regimens to prevent the development of graft-versus-host disease (GVHD) are essential to the success of allogeneic hematopoietic cell transplantation (HCT). After revolutionizing haploidentical transplantation, post-transplantation cyclophosphamide (PTCy) is now being evaluated for HCT performed from related and unrelated donors. In this setting, 2 recent randomized studies have demonstrated lower rates of GVHD and superior GVHD-free, relapse-free survival with PTCy compared with conventional GVHD prophylaxis. The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) is currently conducting a large, randomized phase III, multicenter trial (BMT CTN 1703) comparing PTCy/tacrolimus/mycophenolate mofetil to tacrolimus/methotrexate as GVHD prophylaxis regimens in reduced-intensity allogeneic HCT. Here we review the ongoing study, highlight its importance to the field, and explore the possible implications of its results on clinical practice.
Assuntos
Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Medula Óssea , Ciclofosfamida/uso terapêutico , Doença Enxerto-Hospedeiro/prevenção & controle , Humanos , Estudos Multicêntricos como Assunto , Ácido Micofenólico , Ensaios Clínicos Controlados Aleatórios como Assunto , Condicionamento Pré-Transplante , Doadores não RelacionadosRESUMO
Allogeneic hematopoietic stem cell transplantation (alloHCT) may be associated with significant morbidity and mortality, resulting in increased healthcare utilization (HCU). To date, no multicenter comparative cost analyses have specifically evaluated alloHCT in children with acute leukemia. In this retrospective cohort study, we examined the relationship between survival and HCU while investigating the hypothesis that matched sibling donor (MSD) alloHCT has significantly lower inpatient HCU with unrelated donor (URD) alloHCT, and that among URDs, umbilical cord blood (UCB) alloHCT will have higher initial utilization but lower long-term utilization. Clinical and transplantation outcomes data from the Center for International Blood and Marrow Transplant Research (CIBMTR) were merged with inpatient cost data from the Pediatric Health Information System (PHIS) database using a probabilistic merge methodology. The merged dataset comprised US patients age 1 to 21 years who underwent alloHCT for acute leukemia between 2004 and 2011 with comprehensive CIBMTR data at a PHIS hospital. AlloHCT was analyzed by donor type, with specific analysis of utilization and costs using PHIS claims data. The primary outcomes of overall survival (OS), leukemia-free survival (LFS), and inpatient costs were evaluated using Kaplan-Meier curves and Cox and Poisson models. A total of 632 patients were identified in both the CIBMTR and PHIS data. The 5-year LFS was 60% for MSD alloHCT, 47% for well-matched matched unrelated donor bone marrow (MUD) alloHCT, 48% for mismatched unrelated donor alloHCT, and 45% for UCB alloHCT (P = .09). Total adjusted costs were significantly lower for MSD alloHCT versus MUD alloHCT by day 100 (adjusted cost ratio [ACR], .73; 95% confidence interval [CI], .62 to .86; P < .001), and higher for UCB alloHCT versus MUD alloHCT (ACR, 1.27; 95% CI, 1.11 to 1.45; P < .001). By 2 years, total adjusted costs remained significantly lower for MSD alloHCT compared with MUD alloHCT (ACR, .67; 95% CI, .56 to .81; P < .001) and higher for UCB alloHCT compared with MUD alloHCT (ACR, 1.25; 95% CI, 1.02 to 1.52; P = .0280). Our data show that UCB and MUD alloHCT provide similar survival outcomes; however, MUD alloHCT has a significant advantage in cost by day 100 and 2 years. More research is needed to determine whether the cost difference among URD alloHCT approaches remains significant with a larger sample size and/or beyond 2 years post-alloHCT.
Assuntos
Sistemas de Informação em Saúde , Transplante de Células-Tronco Hematopoéticas , Leucemia Mieloide Aguda , Adolescente , Adulto , Criança , Pré-Escolar , Humanos , Lactente , Estudos Retrospectivos , Doadores não Relacionados , Adulto JovemRESUMO
Cytogenetic risk stratification at diagnosis has long been one of the most useful tools to assess prognosis in acute lymphoblastic leukemia (ALL). To examine the prognostic impact of cytogenetic abnormalities on outcomes after allogeneic hematopoietic cell transplantation, we studied 1731 adults with Philadelphia-negative ALL in complete remission who underwent myeloablative or reduced intensity/non-myeloablative conditioning transplant from unrelated or matched sibling donors reported to the Center for International Blood and Marrow Transplant Research. A total of 632 patients had abnormal conventional metaphase cytogenetics. The leukemia-free survival and overall survival rates at 5 years after transplantation in patients with abnormal cytogenetics were 40% and 42%, respectively, which were similar to those in patients with a normal karyotype. Of the previously established cytogenetic risk classifications, modified Medical Research Council-Eastern Cooperative Oncology Group score was the only independent prognosticator of leukemia-free survival (P=0.03). In the multivariable analysis, monosomy 7 predicted post-transplant relapse [hazard ratio (HR)=2.11; 95% confidence interval (95% CI): 1.04-4.27] and treatment failure (HR=1.97; 95% CI: 1.20-3.24). Complex karyotype was prognostic for relapse (HR=1.69; 95% CI: 1.06-2.69), whereas t(8;14) predicted treatment failure (HR=2.85; 95% CI: 1.35-6.02) and overall mortality (HR=3.03; 95% CI: 1.44-6.41). This large study suggested a novel transplant-specific cytogenetic scheme with adverse [monosomy 7, complex karyotype, del(7q), t(8;14), t(11;19), del(11q), tetraploidy/near triploidy], intermediate (normal karyotype and all other abnormalities), and favorable (high hyperdiploidy) risks to prognosticate leukemia-free survival (P=0.02). Although some previously established high-risk Philadelphia-negative cytogenetic abnormalities in ALL can be overcome by transplantation, monosomy 7, complex karyotype, and t(8;14) continue to pose significant risks and yield inferior outcomes.
Assuntos
Transplante de Células-Tronco Hematopoéticas , Leucemia Mieloide Aguda , Leucemia-Linfoma Linfoblástico de Células Precursoras , Adulto , Aberrações Cromossômicas , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Leucemia-Linfoma Linfoblástico de Células Precursoras/diagnóstico , Leucemia-Linfoma Linfoblástico de Células Precursoras/genética , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Estudos Retrospectivos , Condicionamento Pré-Transplante , Transplante HomólogoRESUMO
BACKGROUND: Financial distress from medical treatment is an increasing concern. Healthcare organizations may have different levels of organizational commitment, existing programs, and expected outcomes of screening and management of patient financial distress. PATIENTS AND METHODS: In November 2018, representatives from 17 (63%) of the 27 existing NCCN Member Institutions completed an online survey. The survey focused on screening and management practices for patient financial distress, perceived barriers in implementation, and leadership attitudes about such practices. Due to the lack of a validated questionnaire in this area, survey questions were generated after a comprehensive literature search and discussions among the study team, including NCCN Best Practices Committee representatives. RESULTS: Responses showed that 76% of centers routinely screened for financial distress, mostly with social worker assessment (94%), and that 56% screened patients multiple times. All centers offered programs to help with drug costs, meal or gas vouchers, and payment plans. Charity care was provided by 100% of the large centers (≥10,000 unique annual patients) but none of the small centers that responded (<10,000 unique annual patients; P=.008). Metrics to evaluate the impact of financial advocacy services included number of patients assisted, bad debt/charity write-offs, or patient satisfaction surveys. The effectiveness of institutional practices for screening and management of financial distress was reported as poor/very poor by 6% of respondents. Inadequate staffing and resources, limited budget, and lack of reimbursement were potential barriers in the provision of these services. A total of 94% agreed with the need for better integration of financial advocacy into oncology practice. CONCLUSIONS: Three-fourths of NCCN Member Institutions reported screening and management programs for financial distress, although the actual practices and range of services vary. Information from this study can help centers benchmark their performance relative to similar programs and identify best practices in this area.
Assuntos
Status Econômico , Financiamento Pessoal , Gastos em Saúde , Neoplasias , Atenção à Saúde/economia , Humanos , Oncologia , Neoplasias/diagnóstico , Neoplasias/economia , Neoplasias/terapia , Inquéritos e QuestionáriosRESUMO
Family caregivers are essential partners for patients undergoing hematopoietic cell transplantation (HCT). The caregiving role is emotionally, physically, and financially demanding. Intervention efforts to provide relief for caregiver stress during HCT are highly warranted. Storytelling interventions are accruing evidence for efficacy in therapeutic contexts. The purpose of this study was to conduct a 3-full consecutive day digital storytelling (DST) workshop to build knowledge on caregivers' lived experiences during HCT, to pilot test DST with a small group of HCT caregivers, and to demonstrate feasibility and acceptability using qualitative and quantitative measures. Six adult caregivers of allogeneic HCT recipients (mean age, 60.2 years) attended a 3-day DST program (66% female, 83% white). All successfully created their personal audiovisual digital story (2 to 3 minutes long) and completed a survey. All participants rated the DST workshop as highly acceptable and therapeutic (mean score 5, on a scale of 1 to 5). Group discussions and interviews with participants further demonstrated high satisfaction and acceptability of the workshop format, setting, process, and structure. The survey results showed decreases in anxiety and depression from before to after the DST workshop with all participants showing change in the expected direction. This study demonstrates the feasibility and acceptability of a 3-day DST workshop as a distress-relieving tool for HCT caregivers. Future research is needed to test the efficacy of DST relative to a control condition.