{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,25]],"date-time":"2026-08-25T15:31:00Z","timestamp":1787671860732,"version":"build-2736575974"},"reference-count":28,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2026,7,25]],"date-time":"2026-07-25T00:00:00Z","timestamp":1784937600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,7,25]],"date-time":"2026-07-25T00:00:00Z","timestamp":1784937600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp;amp; Cancer"],"published-print":{"date-parts":[[2026,10]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Sickle cell disease (SCD) is a highly morbid condition resulting in poor quality of life and early death. Gene therapy offers opportunity for cure, but its resource\u2011 and time\u2011intensive nature may limit access.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Procedure<\/jats:title>\n                    <jats:p>We conducted a single\u2010center, cross\u2010sectional study to determine the proportion of families with a child with SCD who report the ability to access a hypothetical curative therapy and to identify family\u2010reported resource barriers. A single\u2010timepoint survey was administered to 100 families with children aged 2\u201317 years receiving sickle cell care at a quaternary care center. The survey queried parent\u2010reported household characteristics and unmet basic needs in food, housing, utilities, or transportation (household material hardship [HMH]) and assessed parent\u2010reported ability to access a hypothetical curative treatment based on a vignette. Multivariable logistic regression examined associations of HMH and disease severity with family\u2010reported access.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Of the evaluable 82 families, 60% experienced HMH over the prior 12 months despite robust social work involvement (78%) and participation in means\u2010tested benefits (59%). Forty percent of parents reported an ability to obtain curative therapy with current resources. Upon adjustment for key covariates, HMH exposure was independently associated with 95% lower odds of family\u2010reported ability to obtain curative therapy.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Although gene therapy offers a potential cure for SCD, fewer than half of families reported ability to access it with current supports. HMH exposure affected nearly two\u2010thirds of families and was independently associated with lower odds of access. As highly centralized, resource\u2010intensive treatments expand, resource\u2010targeted interventions are needed to ensure equitable access and receipt.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/1545-5017.70536","type":"journal-article","created":{"date-parts":[[2026,7,25]],"date-time":"2026-07-25T07:32:16Z","timestamp":1784964736000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Out of Reach: Understanding Resource Barriers to Gene Therapy for Pediatric Patients With Sickle Cell Disease"],"prefix":"10.1002","volume":"73","author":[{"given":"Colleen A.","family":"Kelly","sequence":"first","affiliation":[{"name":"Dana\u2010Farber\/Boston Children's Cancer and Blood Disorders Center Harvard Medical School  Boston Massachusetts USA"},{"name":"Division of Population Science Dana\u2010Farber Cancer Institute  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Morgan A.","family":"Paul","sequence":"additional","affiliation":[{"name":"Department of Data Science Dana\u2010Farber Cancer Institute  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"McKenzie","family":"Griffin","sequence":"additional","affiliation":[{"name":"Dana\u2010Farber\/Boston Children's Cancer and Blood Disorders Center Harvard Medical School  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sunyu","family":"Kang","sequence":"additional","affiliation":[{"name":"Dana\u2010Farber\/Boston Children's Cancer and Blood Disorders Center Harvard Medical School  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4753-5144","authenticated-orcid":false,"given":"Rahela","family":"Aziz\u2010Bose","sequence":"additional","affiliation":[{"name":"Dana\u2010Farber\/Boston Children's Cancer and Blood Disorders Center Harvard Medical School  Boston Massachusetts USA"},{"name":"Division of Population Science Dana\u2010Farber Cancer Institute  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leanne","family":"Duhaney","sequence":"additional","affiliation":[{"name":"Department of Cardiology Boston Children's Hospital  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Callie","family":"Fry","sequence":"additional","affiliation":[{"name":"Dana\u2010Farber\/Boston Children's Cancer and Blood Disorders Center Harvard Medical School  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7587-4875","authenticated-orcid":false,"given":"Puja J.","family":"Umaretiya","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Texas Southwestern Medical Center  Dallas Texas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristine","family":"Karvonen","sequence":"additional","affiliation":[{"name":"Public Health Sciences Division Fred Hutchinson Cancer Center  Seattle Washington USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erica","family":"Esrick","sequence":"additional","affiliation":[{"name":"Dana\u2010Farber\/Boston Children's Cancer and Blood Disorders Center Harvard Medical School  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sharl","family":"Azar","sequence":"additional","affiliation":[{"name":"Massachusetts General Hospital Harvard Medical School  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natasha M.","family":"Archer","sequence":"additional","affiliation":[{"name":"Dana\u2010Farber\/Boston Children's Cancer and Blood Disorders Center Harvard Medical School  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kira","family":"Bona","sequence":"additional","affiliation":[{"name":"Dana\u2010Farber\/Boston Children's Cancer and Blood Disorders Center Harvard Medical School  Boston Massachusetts USA"},{"name":"Division of Population Science Dana\u2010Farber Cancer Institute  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,7,25]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2022.10233"},{"key":"e_1_2_13_3_1","volume-title":"Sickle Cell Disease: Milestones in Research and Clinical Progress","author":"National Heart, Lung, and Blood 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