OPTN/SRTR 2021 Annual Data Report: Lung.

The number of lung transplants has continued to decline since 2020, a period that coincides with the onset of the COVID-19 pandemic. Lung allocation policy continues to undergo considerable change in preparation for adoption of the Composite Allocation Score system in 2023, beginning with multiple adaptations to the calculation of the Lung Allocation Score that occurred in 2021. The number of candidates added to the waiting list increased after a decline in 2020, while waitlist mortality has increased slightly with a decreased number of transplants. Time to transplant continues to improve, with 38.0% of candidates waiting fewer than 90 days for a transplant. Posttransplant survival remains stable, with 85.3% of transplant recipients surviving to 1 year; 67%, to 3 years; and 54.3%, to 5 years.

Does anybody really know what (the kidney median waiting) time is?

The median waiting time (MWT) to deceased donor kidney transplant is of interest to patients, clinicians, and the media but remains elusive due to both methodological and philosophical challenges. We used Organ Procurement and Transplantation Network data from January 2003 to March 2022 to estimate MWTs using various methods and timescales, applied overall, by era, and by candidate demographics. After rising for a decade, the overall MWT fell to 5.19 years between 2015 and 2018 and declined again to 4.05 years (April 2021 to March 2022), based on the Kaplan-Meier method applied to period-prevalent cohorts. MWTs differed markedly by blood type, donor service area, and pediatric vs adult status, but to a lesser degree by race/ethnicity. Choice of methodology affected the magnitude of these differences. Instead of waiting years for an answer, reliable kidney MWT estimates can be obtained shortly after a policy is implemented using the period-prevalent Kaplan-Meier approach, a theoretical but useful construct for which we found no evidence of bias compared with using incident cohorts. We recommend this method be used complementary to the competing risks approach, under which MWT is often inestimable, to fill the present information void concerning the seemingly simple question of how long it takes to get a kidney transplant in the United States.

A Qualitative Analysis of State Medicaid Coverage Benefits for Allogeneic Hematopoietic Cell Transplantation (alloHCT) for Patients with Sickle Cell Disease (SCD).

Sickle cell disease (SCD) is the most common inherited hemoglobin disorder, affecting approximately 100,000 people in the United States. Allogeneic hematopoietic cell transplantation (alloHCT), also known as bone marrow transplant (BMT), is currently the only established curative option for SCD. However, alloHCT is an optional benefit under Medicaid. This study of alloHCT coverage for patients with SCD aims to understand the scope of state Medicaid coverage benefits and BMT financial coordinators' experience working with their state Medicaid programs. States estimated to have more than 50 newborns diagnosed with SCD in 2016 and at least one active BMT Clinical Trials Network (1503 [STRIDE 2], NCT02766465) transplant center (TC) were eligible to participate in this study. Qualitative, semi-structured interviews 30 to 60 minutes in length were conducted with BMT financial coordinators via telephone between May and October 2019. A total of 10 BMT financial coordinators from 10 TCs representing eight states (Florida, Georgia, Illinois, Michigan, New York, Pennsylvania, Texas, and Virginia) participated in the semi-structured interviews. Coordinators in all of the included states reported that alloHCT in children with SCD with a human leukocyte antigen-matched sibling donor was covered by their state Medicaid programs. However, only two states (Florida and Texas) had legislative policies mandating coverage of routine medical costs for patients in clinical trials. TCs in two states (Illinois and Pennsylvania) reported accepting out-of-state Medicaid insurance, but only one state (Michigan) covered both travel and lodging for the patient and one caregiver. Four themes emerged when coordinators were asked about their perspectives and experiences working with their corresponding state Medicaid programs: (1) state Medicaid eligibility criteria based on disability were perceived as being restrictive, and Medicaid reimbursement rates were reported to be low; (2) Medicaid fee-for-service plans were perceived as being more comprehensive and easier to navigate compared to comprehensive managed care (CMC) plans; (3) there is a need to address caregiver and financial assistance beyond the health care costs; and (4) completing the insurance authorization process leading up to alloHCT is critical, including peer-to-peer reviews. There is limited legislative policy to help ensure access to clinical trials and provide out-of-state benefits and travel and lodging for Medicaid enrollees with SCD. These data provide insight into potential areas that could influence changes in policy to enhance access to curative therapy for SCD.

Hematopoietic Cell Transplantation Outcomes among Medicaid and Privately Insured Patients with Sickle Cell Disease.

Compared with privately insured patients, recipients of Medicaid have been reported to have worse outcomes in several clinical conditions and following various surgical and medical procedures. However, the relationship between health insurance status and allogeneic hematopoietic cell transplantation (alloHCT) outcomes among patients with sickle cell disease (SCD) is not well described. We sought to compare alloHCT outcomes between patients with SCD who underwent alloHCT while enrolled on Medicaid versus those who underwent alloHCT while covered by private health insurance. We conducted a retrospective multicenter study using data reported to the Center for International Blood and Marrow Transplant Research. US patients enrolled on Medicaid or private insurance who underwent a first alloHCT for SCD between 2008 and 2018 were eligible for this study. The primary outcome was event-free survival (EFS), defined as time to death or graft failure. Secondary outcomes included overall survival (OS), graft failure, acute graft-versus-host disease (GVHD), and chronic GVHD. Univariate analysis was performed using the Kaplan-Meier method for EFS and OS. The proportion of patients with graft failure, acute GVHD, and/or chronic GVHD was calculated using the cumulative incidence estimator to accommodate competing risks (ie, death). Cox regression was used to identify factors associated with EFS, OS, graft failure, and acute and chronic GVHD. A total of 399 patients (Medicaid, n = 225; private insurance, n = 174) were included in this study. The median duration of follow-up was 34 months (range, 1.0 to 134.7 months) for the Medicaid group and 38.7 months (range, 0.3 to 139.3 months) for the private insurance group. Compared with the patients with private insurance, those on Medicaid had a significantly lower 3-year EFS (75.4% [95% confidence interval (CI), 69.4% to 81%] versus 82.2% [95% CI, 76.9% to 87.8%]; P = .0279) and a significantly higher 3-year cumulative incidence of graft failure (17.2% [95% CI, 12.5% to 22.5%] versus 10.5% [95% CI, 6.4% to 15.4%]; P = .0372). There were no significant between-group differences in 3-year OS (P = .6337) or in the cumulative incidence of acute GVHD (P = .4556) or chronic GVHD (P = .6878). Cox regression analysis after adjusting for other significant variables showed that the patients enrolled on Medicaid had a lower EFS (hazard ratio [HR], 2.36; 95% CI, 1.44 to 3.85; P = .0006) and a higher cumulative incidence of graft failure (HR, 2.57; 95% CI, 1.43 to 4.60; P = .0015), with no significant between-group differences in OS (HR, 0.99; 95% CI, 0.47 to 2.07; P = .9765), acute GVHD (HR, 0.94; 95% CI, 0.59 to 1.49; P = .7905), or cGVHD (HR, 0.98; 95% CI, 0.65 to 1.48; P = .9331). That EFS is worse in patients on Medicaid compared with privately insured individuals following alloHCT for SCD provides the rationale for research to better understand the mechanisms by which insurance status impacts alloHCT outcomes among patients with SCD.

Hematopoietic Cell Transplantation: Practice Predictions for the Year 2023.

Research priorities are best determined by the most pressing scientific questions, in the context of current knowledge. However, definitive research studies take time, while real-world experience accumulates. Adoption of new practices before adequate comparison with current treatments threatens successful study conduct and may expose patients to what ultimately turns out to be inferior treatment. We conducted a survey to understand the hematopoietic cell transplantation (HCT) community's predictions about future practice trends in the HCT field and results of ongoing Blood and Marrow Transplant Clinical Trials Network (BMT CTN) trials to gauge how the HCT community views the treatments being studied. The survey was distributed between February and March 2019 to an electronic mailing list of HCT clinicians practicing in the United States maintained by the Center for International Blood and Marrow Transplant Research (CIBMTR). Of 986 clinicians surveyed, 315 responded (32%). They predicted an increase in the number of HCTs performed for malignant hematologic diseases and benign diseases such as sickle cell, autoimmune, and genetic disorders. The majority (63%) predicted that matched related donors will remain the preferred donor source for adult HCT recipients in 2023, but 21% predicted haploidentical (haplo) donors and 17% predicted matched unrelated donors would be the preferred source. Most respondents (65%) predicted a decrease in the use of umbilical cord blood (UCB) as a graft source for HCT. Most respondents also predicted that calcineurin-based graft-versus-host disease (GVHD) prophylaxis would be replaced by post-transplantation cyclophosphamide (PTCy) (55%), biomarker use would become standard practice to guide GVHD therapy (73%), and steroids would be combined with other agents as first-line therapy for newly diagnosed acute (53%) and chronic GVHD (54%). In ongoing BMT CTN trials in which outcomes are not yet known, 60% to 92% of respondents had an opinion about which arm they thought would be superior. However, not all respondents predicted the same outcome, with 44% to 88% choosing the same arm. There was no clear relationship between the proportion predicting the same arm would win and accrual to the trial. Survey respondents were optimistic about an increasing volume of transplantation procedures, and they also expected significant changes in HCT practice over the next few years, including wider adoption of PTCy GVHD prophylaxis, increased use of biomarkers to guide GVHD therapy, and decreased use of UCB HCT. The degree of equipoise in the community about the relative efficacy of therapies being studied did not seem to affect accrual to current BMT CTN trials, but this is an area that needs further investigation.

Healthcare Costs of Treating Privately Insured Patients with Acute Myeloid Leukemia in the United States from 2004 to 2014: A Generalized Additive Modeling Approach.

OBJECTIVES: The primary objective of this study was to predict healthcare cost trajectories for patients with newly diagnosed acute myeloid leukemia (AML) receiving allogeneic hematopoietic cell transplantation (alloHCT), as a function of days since chemotherapy initiation, days relative to alloHCT, and days before death or last date of insurance eligibility (LDE). An exploratory objective examined patients with AML receiving chemotherapy only. METHODS: We used Optum's de-identified Clinformatics® Data Mart Database to construct cumulative cost trajectories from chemotherapy initiation to death or LDE (through 31 December 2014) for US patients aged 20-74 years diagnosed between 1 March 2004 and 31 December 2013 (n = 187 alloHCT; n = 253 chemotherapy only). We used generalized additive modeling (GAM) to predict expected trajectories and bootstrapped confidence intervals (CIs) at user-specified intervals conditional on dates of alloHCT and death or LDE relative to chemotherapy initiation. RESULTS: Expected costs (in 2017 values) for a hypothetical patient receiving alloHCT 60 days after chemotherapy initiation and followed for 5 years were $US572,000 (95% CI 517,000-633,000); $US119,000 (95% CI 51,000-192,000); $US102,000 (95% CI 0-285,000); $US79,000 (95% CI 0-233,000), for years 1-4, respectively, and either $US494,000 (95% CI 212,000-799,000) or $US108,000 (95% CI 0-230,000) in year 5, whether the patient died or was lost to follow-up on day 1825, respectively. CONCLUSIONS: Rates of cost accrual varied over time since chemotherapy initiation, with accelerations around the time of alloHCT and death. GAM is a potentially useful approach for imputing longitudinal costs relative to treatment initiation and one or more intercurrent, clinical, or terminal events in randomized controlled trials or registries with unrecorded costs or for dynamic decision-analytic models.

Urgent Time to Allogeneic Hematopoietic Cell Transplantation: A National Survey of Transplant Physicians and Unrelated Donor Search Coordinators Facilitated by the Histocompatibility Advisory Group to the National Marrow Donor Program.

To characterize donor search and selection practices, the National Marrow Donor Program (NMDP) Histocompatibility Advisory Group developed a survey of allogeneic hematopoietic cell transplant (HCT) physicians and search coordinators. The objectives were to describe search practices, understand practices surrounding urgent time to HCT, and characterize strategies used when identifying a matched unrelated donor is unlikely. Participants included US physician members of the American Society for Transplantation and Cellular Therapy and donor search coordinators within the NMDP network. The web-based survey was conducted from February to May 2018. Three hundred seventeen of 858 physicians (37%) and 225 of 327 coordinators (69%) responded, of which 263 and 194, respectively, were eligible and included in the analysis. Most centers, 142 (95%), were represented; 108 (72%) had at least 1 physician and 128 (85%) had at least 1 coordinator respondent. Most (68% physicians, 61% coordinators) indicated donor selection decisions were made by individual physicians. Urgent time to HCT was most commonly (90% and 87% of physicians and coordinators, respectively) defined as HCT within 4 to 6 weeks of search initiation. Higher HCT urgency was associated with a higher disease risk index. For urgent cases with low probability of an 8/8 matched unrelated donor , 75% and 80% of physicians and coordinators endorsed a short (1 to 2 weeks) unrelated donor search before proceeding to an alternative donor source. NMDP-provided solutions to expedite donor identification were strongly endorsed. This survey clarified current donor selection practices in the United States and defined urgent time to HCT. These data provide insight to NMDP on potential solutions to support the path to transplant, such as highlighting futile searches and providing alternative donor options at the time of search initiation.

Factors Influencing Efficacy of Nutrition Education Interventions: A Systematic Review.

OBJECTIVE: To examine systematically factors that contribute to the efficacy of nutrition education interventions in promoting behavior change for good health based on their stated objective. In a departure from previous reviews, the researchers investigated factors that lead to success of various types of interventions. Critical analysis of these factors constituted the outcome of this review. METHODS: This study followed Preferred Reporting Items for Systematic Reviews and Meta-analysis criteria. A total of 246 original articles published between 2009 and 2015 in PubMed, Medline, Web of Science, Academic Search Complete, Science Direct, Cochrane Reviews, ERIC, and PsychLIT were initially considered. The number was screened and scaled down to 40 publications for the final analysis. Quality assessment was based on the Cochrane Handbook for Systematic Reviews of Intervention. Studies were rated as having low risk of bias, moderate risk, or high risk. RESULTS: Efficacy of nutrition education interventions depended on major factors: interventions that lasted ≥5 months; having ≤3 focused objectives; appropriate design and use of theories; fidelity in interventions; and support from policy makers and management for worksite environmental interventions. CONCLUSIONS AND IMPLICATIONS: Intervention duration of ≥5 months, ≤3 focused objectives, randomization, use of theories, and fidelity are factors that enhance success of interventions based on the results of this study.

Prevalence of Food Insecurity in Low-Income Neighborhoods in West Texas.

OBJECTIVE: To determine the prevalence of food insecurity and the coping strategies and to investigate the role of safety nets among low-income households in urban and rural west Texas. DESIGN: The Core Food Security Module, an 18-item scale, was used in a cross-sectional purposeful convenience sample comparing rural and urban households, whereas the demographic survey assessed participation in food assistance/safety net programs. SETTING: Rural and urban neighborhoods in west Texas. PARTICIPANTS: Sample size of 191 participants from low-income households, predominantly African American and Hispanic people. MAIN OUTCOMES MEASURES: Levels of food insecurity and use of safety nets. ANALYSES: Comparisons between rural and urban households and between food-secure and food-insecure households were analyzed using the chi-square test of independence for categorical variables. Fisher's exact test was used whenever the number in each cell was < 5 in 2 × 2 contingency tables. RESULTS: Prevalence of household and child food insecurity in west Texas was 63% and 43%, respectively. Forgoing balanced meals was a common coping strategy. There was high intake of affordable energy-intense foods. CONCLUSIONS AND IMPLICATIONS: The high prevalence of food insecurity in low-income households in west Texas led to high intake of energy-intense food with low nutrients, resulting in higher prevalence of anemia, obesity, and other chronic diseases. There was low participation in safety net programs. Educational interventions on food choices are recommended.

Plasma Concentrations of Hepcidin in Anemic Zimbabwean Infants.

OBJECTIVE: Anemia in infancy is a global public health problem. We evaluated the relative contributions of iron deficiency and inflammation to infant anemia. METHODS: We measured plasma hepcidin, ferritin, soluble transferrin receptor (sTfR), alpha-1-acid glycoprotein and C-reactive protein (CRP) by ELISA on archived plasma from 289 HIV-unexposed anemic or non-anemic Zimbabwean infants at ages 3 mo, 6 mo and 12 mo. Among anemic infants, we determined the proportion with iron-deficiency anemia (IDA) and anemia of inflammation (AI). We undertook regression analyses of plasma hepcidin and anemia status, adjusting for sex, age and birthweight. RESULTS: Anemic infants at 3 mo were more stunted and had higher CRP (median 0.45 vs 0.21 mg/L; P = 0.037) and hepcidin (median 14.7 vs 9.7 ng/mL; P = 0.022) than non-anemic infants, but similar levels of ferritin and sTfR; 11% infants had IDA and 15% had AI. Anemic infants at 6 mo had higher hepcidin (median 7.9 vs 4.5 ng/mL; P = 0.016) and CRP (median 2.33 vs 0.32 mg/L; P<0.001), but lower ferritin (median 13.2 vs 25.1 µg/L; P<0.001) than non-anemic infants; 56% infants had IDA and 12% had AI. Anemic infants at 12 mo had lower ferritin (median 3.2 vs 22.2 µg/L; P<0.001) and hepcidin (median 0.9 vs 1.9 ng/mL; P = 0.019), but similar CRP levels; 48% infants had IDA and 8% had AI. Comparing anemic with non-anemic infants, plasma hepcidin was 568% higher, 405% higher and 64% lower at 3 mo, 6 mo and 12 mo, respectively, after adjusting for sex and birthweight (all p<0.01). Plasma hepcidin declined significantly with age among anemic but not non-anemic infants. Girls had 61% higher hepcidin than boys, after adjusting for age, anemia and birthweight (p<0.001). CONCLUSION: Anemia is driven partly by inflammation early in infancy, and by iron deficiency later in infancy, with plasma hepcidin concentrations reflecting the relative contribution of each. However, there is need to better characterize the drivers of hepcidin during infancy in developing countries.