Long-term safety and efficacy of gene-corrected autologous keratinocyte grafts for recessive dystrophic epidermolysis bullosa.
Orphanet journal of rare diseases
2022; 17 (1): 377
BACKGROUND: Recessive dystrophic epidermolysis bullosa (RDEB) is a rare, devastating blistering genodermatosis caused by mutations in the COL7A1 gene, which encodes for type VII collagen and is necessary for dermal-epidermal adhesion and integrity. Disease manifestations include severe and debilitating wounds, aggressive squamous cell carcinomas, and premature death; however, there are currently no approved therapies. This Phase 1/2a, open-label study evaluated the long-term efficacy and safety of gene-corrected autologous keratinocyte grafts (EB-101) for chronic RDEB wounds.METHODS: Autologous keratinocytes were harvested from participants with severe RDEB, transduced with a retrovirus containing the full-length COL7A1 gene, and grown into 5*7cm (35 cm2) sheets. Gene-corrected keratinocyte sheets were then transplanted onto chronic RDEB wounds present for≥12weeks.RESULTS: Seven adult participants with severe RDEB were grafted with six sheets each (42 total sheets) onto wounds and followed for a mean of 5.9years (range 4-8years). Long-term improvements in wound healing and symptoms were observed. At year five, 70% (21/30) of treated sites demonstrated≥50% wound healing compared to baseline by investigator global assessment. No sites with≥50% wound healing were painful or pruritic, compared to 67% (6/9) of sites with<50% wound healing (p<0.001) at year five. Grafts were well-tolerated throughout long-term follow-up. No serious adverse events related to treatment were reported over a mean of 5.9years of follow-up. No persistent systemic autoimmunity against type VII collagen or replication-competent retrovirus infections were identified, and no participants developed squamous cell carcinomas related to treatment during long-term follow-up.CONCLUSIONS: Treatment with EB-101 appears safe and efficacious, and produces long-term improvements in wound healing, pain, and itch for RDEB patients. Results from the Phase 3 randomized controlled trial are forthcoming.TRIAL REGISTRATION: ClinicalTrials.gov, NCT01263379. Registered December 15, 2010. https://clinicaltrials.gov/ct2/show/NCT01263379.
View details for DOI 10.1186/s13023-022-02546-9
View details for PubMedID 36253825
A global, cross-sectional survey of patient-reported outcomes, disease burden, and quality of life in epidermolysis bullosa simplex.
Orphanet journal of rare diseases
2022; 17 (1): 270
Epidermolysis bullosa simplex (EBS) comprises a group of rare, blistering genodermatoses. Prior work has been limited by small sample sizes, and much remains unexplored about the disease burden and health-related quality of life (QOL) of patients with EBS. The aim of this study was to characterize the most common patient-reported clinical manifestations and the health-related impact of QOL in EBS, and to examine differences in disease burden by age.Patients with a diagnosis of epidermolysis bullosa (EB) or their caregivers completed a one-time online survey administered by EBCare, an international online EB registry. Survey data from respondents self-reporting a diagnosis of EBS were analyzed for clinical and wound manifestations, medication use, and QOL (using Quality of Life in Epidermolysis Bullosa [QOLEB] scores). Differences across age groups were assessed using Kruskal-Wallis and Fisher's exact tests.There were 214 survey respondents with EBS. The mean age was 32.8 years (standard deviation = 19.2). Many respondents reported blisters (93%), recurrent wounds (89%), pain (74%), chronic wounds (59%), itch (55%), and difficulty walking (44%). Mean QOLEB score was 14.7 (standard deviation = 7.5) indicating a "moderate" impact on QOL, and 12% of respondents required regular use of opiates. Findings were consistent in subgroup analyses restricted to respondents with diagnostic confirmation via genetic testing or skin biopsy (n = 63 of 214). Age-stratified analyses revealed differences in disease burden: younger respondents were more likely to self-report severe disease (24% vs. 19% vs. 5% for respondents aged 0-9 vs. 10-17 vs. 18 + , p = 0.001), failure to thrive (9% vs. 15% vs. 3%, p = 0.02), and use of gastrostomy tubes (15% vs. 12% vs. 1%, p < 0.001) and topical antibiotics (67% vs. 69% vs. 34%, p < 0.001), while older respondents were more likely to be overweight or obese (6% vs. 0% vs. 51%, p < 0.001) and have difficulty walking (24% vs. 46% vs. 48%, p = 0.04).In the largest international cross-sectional survey of EBS patients conducted, respondents reported extensive disease burden including significant wounding, pain, itch, difficulty walking, and impact on QOL. Age stratified disease manifestations. These findings suggest significant unmet need, and treatment and counseling for EBS patients should consider age-specific differences.
View details for DOI 10.1186/s13023-022-02433-3
View details for PubMedID 35841105
Halo Nevi in the Pediatric Population
JOURNAL OF THE DERMATOLOGY NURSES ASSOCIATION
2023; 15 (1): 41-45
View details for DOI 10.1097/JDN.0000000000000719
View details for Web of Science ID 000916888600005
Association of Intraoperative Antibiotic Irrigation With Systemic Contact Dermatitis.
This case series describes the development of morbilliform drug eruption after breast surgery.
View details for DOI 10.1001/jamadermatol.2022.4458
View details for PubMedID 36383358
Chronic Dermatitis Due to Jellyfish Envenomation.
Dermatitis : contact, atopic, occupational, drug
View details for DOI 10.1097/DER.0000000000000930
View details for PubMedID 35943348
Associations between dietary and supplemental vitamin A and skin cancer risk in postmenopausal women
ELSEVIER SCIENCE INC. 2022: S33
View details for Web of Science ID 000829693000370
Assessing the natural history of recessive dystrophic epidermolysis bullosa wounds using a home photography app
ELSEVIER SCIENCE INC. 2022: S131
View details for Web of Science ID 000829693001311
Long-term efficacy and safety of investigational autologous gene-corrected skin sheets (EB-101) for recessive dystrophic epidermolysis bullosa (RDEB)
ELSEVIER SCIENCE INC. 2022: S47
View details for Web of Science ID 000829693000451
Patient-reported disease burden in epidermolysis bullosa simplex (EBS)
ELSEVIER SCIENCE INC. 2022: S33
View details for Web of Science ID 000829693000369
Genotype-phenotype associations in recessive dystrophic epidermolysis bullosa (RDEB)
ELSEVIER SCIENCE INC. 2022: S77
View details for Web of Science ID 000829693001011
Lack of association between dietary and supplemental vitamin E intake with skin cancer risk in postmenopausal women
ELSEVIER SCIENCE INC. 2022: S32
View details for Web of Science ID 000829693000364
New-onset pemphigus vegetans and pemphigus foliaceus following SARS-CoV-2 vaccination: a case series.
JAAD case reports
View details for DOI 10.1016/j.jdcr.2022.07.002
View details for PubMedID 35845348
- Three Cases of Systemic Contact Dermatitis Due to Intraoperative Bacitracin Irrigation During Mastectomy and Breast Reconstruction 2022: e36-e43
- Epidermolysis Bullosa Simplex GeneReviews University of Washington, Seattle. 2022
Histopathologic correlation of skin manifestations of multisystemic inflammatory syndrome in adults (MIS-A) associated with SARS-CoV-2 infection.
JAAD case reports
View details for DOI 10.1016/j.jdcr.2021.06.031
View details for PubMedID 34405113
Geographic and Socioeconomic Disparities in Uveal Melanoma Treatment, 2004-2016
ASSOC RESEARCH VISION OPHTHALMOLOGY INC. 2021
View details for Web of Science ID 000690761100801
Differences in patient perceptions of integrated care among black, hispanic, and white Medicare beneficiaries.
Health services research
OBJECTIVE: This study sought to identify potential disparities among racial/ethnic groups in patient perceptions of integrated care (PPIC) and to explore how methodological differences may influence measured disparities.DATA SOURCE: Data from Medicare beneficiaries who completed the 2015 Medicare Current Beneficiary Survey (MCBS) and were enrolled in Part A benefits for an entire year.STUDY DESIGN: We used 4-point measures of eight dimensions of PPIC and assessed differences in dimensions among racial/ethnic groups. To estimate differences, we applied a "rank and replace" method using multiple regression models in three steps, balancing differences in health status among racial groups and adjusting for differences in socioeconomic status. We reran all analyses with additional SES controls and using standard multiple variable regression.DATA COLLECTION/EXTRACTION METHODS: Not applicable.PRINCIPAL FINDINGS: We found several significant differences in perceived integrated care between Black versus White (three of eight measures) and Hispanic versus White (one of eight) Medicare beneficiaries. On average, Black beneficiaries perceived more integrated support for self-care than did White beneficiaries (mean difference=0.14, SE=0.06, P=.02). Black beneficiaries perceived more integrated specialists' knowledge of past medical history than did White beneficiaries (mean difference=0.12, SE=0.06, P=.01). Black and Hispanic beneficiaries also each reported, on average, 0.18 more integrated medication and home health management than did White beneficiaries (P<.01 and P<.01). These findings were robust to sensitivity analyses and model specifications.CONCLUSIONS: There exist some aspects of care for which Black and Hispanic beneficiaries may perceive greater integrated care than non-Hispanic White beneficiaries. Further studies should test theories explaining why racial/ethnic groups perceive differences in integrated care.
View details for DOI 10.1111/1475-6773.13637
View details for PubMedID 33569775
Socioeconomic Differences Persist in Use of Permanent versus Long-Acting Reversible Contraception: An Analysis of the National Survey for Family Growth, 2006-2010 versus 2015-2017.
OBJECTIVE: Permanent contraception has historically been more prevalent among non-White women with lower education and income. Given increasing popularity of long-acting reversible contraception (LARC), we examine changing sociodemographic patterns of permanent contraception and LARC.STUDY DESIGN: We performed a descriptive analysis of the National Survey of Family Growth (NSFG) from 2006-2017, with multivariable analyses of the 2006-2010 and 2015-2017 cohorts. Using multinomial logistic regression, we investigate predictors of contraceptive category (permanent contraception versus LARC, lower-efficacy contraception versus LARC) in reproductive-aged women.RESULTS: 8,161 respondents were included in two distinct but analogous regression analyses: 1) the most recent survey cohort, 2015-2017, and 2) the cohort a decade prior, 2006-2010. Over this period, the prevalence of LARC increased nearly three-fold (6.2% to 16.7%), while permanent contraception use trended downwards (22% to 18.6%). Yet, in adjusted models, we observed little change in the sociodemographic predictors of permanent contraception: from the early to recent cohort, use of permanent contraception (versus LARC) remained less likely among college graduates (multinomial odds ratio (OR) 0.45[95% CI 0.21, 0.97]) and Hispanic women (OR 0.41[0.21, 0.82]). In addition, high income (>$74,999) and metropolitan residence came to predict less use (OR 0.33[0.13, 0.84] and 0.47[0.23, 0.97]). Multiparity, advanced age (over ≥35), and marital status remained strong predictors of permanent contraception.CONCLUSION: Although use of LARC nearly equals that of permanent contraception in the most recent NSFG survey, socioeconomic differences persist. Continued effort is needed to detect and address structural barriers to accessing the most effective forms of contraception for women.IMPLICATIONS: Comparing 2006-2010 to 2015-2017, reliance on female permanent contraception decreased while LARC use increased, making prevalence more similar. However, significant socioeconomic differences persist in who chooses permanent contraception, with urban, educated, higher-income women more likely to use LARC. Ongoing efforts are needed to understand and reduce economic barriers to LARC.
View details for DOI 10.1016/j.contraception.2020.12.008
View details for PubMedID 33359509
GENDER DIFFERENCES IN MEDICARE BENEFICIARIES' PERCEPTIONS OF INTEGRATED CARE
SPRINGER. 2020: S138
View details for Web of Science ID 000567143600312
Sociodemographic Trends in Long Acting Reversible Contraception vs. Female Sterilization, 2006-2017
LIPPINCOTT WILLIAMS & WILKINS. 2020: 100S
View details for Web of Science ID 000554572900348
Patient Experiences of Integrated Care in Medicare Accountable Care Organizations and Medicare Advantage Versus Traditional Fee-For-Service.
Health insurance design can influence the extent to which clinical care is well-coordinated. Through alternative payment models, Medicare Advantage (MA) and Accountable Care Organizations (ACOs) have the potential to improve integration relative to traditional fee-for-service (FFS) Medicare.To characterize patient experiences of integrated care within Medicare and identify whether MA or ACO beneficiaries perceive greater integration than FFS beneficiaries.Retrospective cross-sectional analysis of the 2015 Medicare Current Beneficiary Survey.Nationally representative sample of 11,978 Medicare beneficiaries.Main outcomes included 8 previously derived domains of patient-perceived integrated care (PPIC), measured on a scale of 1-4.The final sample was 55% female with a mean (SD) age of 71.1 (11.3). In unadjusted analyses, we observed considerable variation across PPIC domains in the full sample, but little variation across subsamples defined by coverage type within a given PPIC domain. In linear models adjusting for a rich set of patient characteristics, we observe no significant benefits of ACOs nor MA relative to FFS, a finding which is robust to alternative specifications and adjustment for multiple comparisons. We similarly observed no benefits in subgroup analyses restricted to states with relatively high market penetration of ACOs or MA.Despite characteristics of ACOs and MA that theoretically promote integrated care, we find that PPIC is largely similar across coverage types in Medicare.
View details for DOI 10.1097/MLR.0000000000001463
View details for PubMedID 33273291
Nonmydriatic Photographic Screening for Diabetic Retinopathy in Pregnant Patients with Pre-Existing Diabetes in a Safety Net Population
Women's Health Reports
2020; 1 (1): 436-443
View details for DOI 10.1089/whr.2020.0082