{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,19]],"date-time":"2026-09-19T21:16:56Z","timestamp":1789852616931,"version":"4.0.1"},"reference-count":79,"publisher":"American Society of Hematology","issue":"7","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2026,8,13]]},"abstract":"<jats:sec>\n                    <jats:title>Abstract<\/jats:title>\n                    <jats:p>Nonmyeloablative-related haploidentical allogeneic stem cell transplantation (NMAC-HID allo-HSCT) has emerged as an additional treatment to achieve durable remission in sickle cell disease (SCD), a prevalent blood disorder characterized by painful vaso-occlusive crises and chronic anemia. The standard of care (SOC) for SCD includes hydroxyurea, pain management, and blood transfusion, but patients with SCD still lose several decades of life expectancy. Gene therapy (GT) for SCD is the other treatment for lifelong disease amelioration in SCD, with accessibility limited by cost and manufacturing capacity in the United States and globally. Two recent prospective studies that evaluated NMAC-HID allo-HSCT validated haploidentical allotransplantation as an efficacious and accessible treatment option in the era of GT. Given the ongoing price negotiation across jurisdictions for GT implementation and the absence of cost-effectiveness data comparing NMAC-HID allo-HSCT and GT, we conducted a cost-effectiveness analysis of NMAC-HID allo-HSCT vs GT vs SOC for adults and children living with SCD. The primary outcomes were the incremental cost-effectiveness ratio and the net monetary benefits across these 3 strategies. The secondary outcome was the maximum cost-effective threshold price for GT compared with NMAC-HID allo-HSCT. Treatment with SOC, NMAC-HID allo-HSCT, and GT accrued 14.3, 20.1, and 22.1 quality-adjusted life-years at costs of $1.22 million, $1.15 million, and $2.75 million, respectively. NMAC-HID allo-HSCT was the cost-effective strategy compared with GT in 100% of 10\u2009000 Monte Carlo iterations across the base case and all scenario analyses. The maximum cost-effective thresholds for GT vs SOC were $1.4 million in the United States and $4200 to $22\u2009000 across India, Nigeria, and Tanzania, depending on willingness-to-pay thresholds.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1182\/blood.2025032290","type":"journal-article","created":{"date-parts":[[2026,6,2]],"date-time":"2026-06-02T14:19:55Z","timestamp":1780409995000},"page":"896-909","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":2,"title":["Haploidentical transplant, gene therapy, and standard care in sickle cell disease: a cost-effectiveness analysis"],"prefix":"10.1182","volume":"148","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4974-1099","authenticated-orcid":false,"given":"Karthik","family":"Chetlapalli","sequence":"first","affiliation":[{"name":"1Yale School of Medicine, New Haven, CT"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4430-1860","authenticated-orcid":false,"given":"Satoko","family":"Ito","sequence":"additional","affiliation":[{"name":"2Section of Medical Oncology and Hematology, Department of Internal Medicine, Yale School of Medicine and Yale Cancer Center, New Haven, CT"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0754-7901","authenticated-orcid":false,"given":"Ding Quan","family":"Ng","sequence":"additional","affiliation":[{"name":"2Section of Medical Oncology and Hematology, Department of Internal Medicine, Yale School of Medicine and Yale Cancer Center, New Haven, CT"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2939-5400","authenticated-orcid":false,"given":"Manraj","family":"Sra","sequence":"additional","affiliation":[{"name":"3Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9336-4123","authenticated-orcid":false,"given":"Daniel","family":"Wang","sequence":"additional","affiliation":[{"name":"1Yale School of Medicine, New Haven, CT"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2046-127X","authenticated-orcid":false,"given":"Harlan M.","family":"Krumholz","sequence":"additional","affiliation":[{"name":"4Section of Cardiovascular Medicine, Department of Internal Medicine, Yale School of Medicine, New Haven, CT"},{"name":"5Yale Center for Outcomes Research and Evaluation, Yale New Haven Hospital and Yale University, New Haven, CT"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3242-0037","authenticated-orcid":false,"given":"Lakshmanan","family":"Krishnamurti","sequence":"additional","affiliation":[{"name":"6Section of Pediatric Hematology, Oncology and Bone Marrow Transplant, Yale School of Medicine, New Haven, CT"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ankur","family":"Pandya","sequence":"additional","affiliation":[{"name":"7Department of Health Policy and Management, Harvard T.H. Chan School of Public Health, Boston, MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1624-4427","authenticated-orcid":false,"given":"George","family":"Goshua","sequence":"additional","affiliation":[{"name":"2Section of Medical Oncology and Hematology, Department of Internal Medicine, Yale School of Medicine and Yale Cancer Center, New Haven, CT"},{"name":"5Yale Center for Outcomes Research and Evaluation, Yale New Haven Hospital and Yale University, New Haven, CT"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","reference":[{"issue":"1","key":"2026081311214223200_bib1","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1001\/jama.2022.10233","article-title":"Sickle cell disease: a review","volume":"328","author":"Kavanagh","year":"2022","journal-title":"JAMA"},{"issue":"10","key":"2026081311214223200_bib2","doi-asserted-by":"crossref","first-page":"1565","DOI":"10.3390\/jcm8101565","article-title":"Allogeneic hematopoietic stem cell transplantation for adults with sickle cell disease","volume":"8","author":"Saraf","year":"2019","journal-title":"J Clin Med"},{"key":"2026081311214223200_bib3","unstructured":"Pfizer Voluntarily Withdraws All Lots of Sickle Cell Disease Treatment OXBRYTA\u00ae (Voxelotor) From Worldwide Markets. 2024. Pfizer Inc. Accessed 26 May 2026. https:\/\/www.pfizer.com\/news\/press-release\/press-release-detail\/pfizer-voluntarily-withdraws-all-lots-sickle-cell-disease."},{"key":"2026081311214223200_bib4","unstructured":"European Commission (EC) Adopts Decision Endorsing CHMP Recommendation to Revoke the Conditional Marketing Authorization for Adakveo\u00ae (Crizanlizumab). 2023. Novartis AG. Accessed 26 May 2026. https:\/\/www.novartis.com\/news\/european-commission-ec-adopts-decision-endorsing-chmp-recommendation-revoke-conditional-marketing-authorization-adakveo-crizanlizumab."},{"issue":"4","key":"2026081311214223200_bib5","doi-asserted-by":"crossref","first-page":"e248","DOI":"10.1016\/S2352-3026(24)00384-3","article-title":"Crizanlizumab with or without hydroxyurea in patients with sickle cell disease (STAND): primary analyses from a placebo-controlled, randomised, double-blind, phase 3 trial","volume":"12","author":"Abboud","year":"2025","journal-title":"Lancet Haematol"},{"issue":"8","key":"2026081311214223200_bib6","doi-asserted-by":"crossref","first-page":"e556","DOI":"10.1016\/S2352-3026(24)00175-3","article-title":"Dismantling cost and infrastructure barriers to equitable access to gene therapies for sickle cell disease","volume":"11","author":"Sharma","year":"2024","journal-title":"Lancet Haematol"},{"issue":"26","key":"2026081311214223200_bib7","doi-asserted-by":"crossref","first-page":"2693","DOI":"10.1182\/blood.2024024519","article-title":"How I treat sickle cell disease with gene therapy","volume":"144","author":"Sharma","year":"2024","journal-title":"Blood"},{"issue":"25","key":"2026081311214223200_bib8","doi-asserted-by":"crossref","first-page":"2654","DOI":"10.1182\/blood.2023023301","article-title":"An international learning collaborative phase 2 trial for haploidentical bone marrow transplant in sickle cell disease","volume":"143","author":"Kassim","year":"2024","journal-title":"Blood"},{"issue":"3","key":"2026081311214223200_bib9","doi-asserted-by":"crossref","first-page":"EVIDoa2400192","DOI":"10.1056\/EVIDoa2400192","article-title":"Haploidentical bone marrow transplantation for sickle cell disease","volume":"4","author":"Kassim","year":"2025","journal-title":"NEJM Evid"},{"issue":"1","key":"2026081311214223200_bib10","doi-asserted-by":"crossref","first-page":"532","DOI":"10.1182\/hematology.2023000486","article-title":"The range of haploidentical transplant protocols in sickle cell disease: all haplos are not created equally","volume":"2023","author":"Kassim","year":"2023","journal-title":"Hematol Am Soc Hematol Educ Program"},{"issue":"22","key":"2026081311214223200_bib11","doi-asserted-by":"crossref","first-page":"4285","DOI":"10.1182\/blood-2012-07-438408","article-title":"HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease","volume":"120","author":"Bolanos-Meade","year":"2012","journal-title":"Blood"},{"issue":"6","key":"2026081311214223200_bib12","doi-asserted-by":"crossref","first-page":"1197","DOI":"10.1016\/j.bbmt.2018.11.027","article-title":"Haploidentical bone marrow transplantation with post-transplantation cyclophosphamide plus thiotepa improves donor engraftment in patients with sickle cell anemia: results of an international learning collaborative","volume":"25","author":"de la Fuente","year":"2019","journal-title":"Biol Blood Marrow Transpl"},{"issue":"5","key":"2026081311214223200_bib13","doi-asserted-by":"crossref","first-page":"335","DOI":"10.1177\/2040620713483063","article-title":"Hematopoietic stem-cell transplantation for sickle cell disease: current evidence and opinions","volume":"4","author":"Shenoy","year":"2013","journal-title":"Ther Adv Hematol"},{"issue":"2","key":"2026081311214223200_bib14","doi-asserted-by":"crossref","first-page":"155","DOI":"10.7326\/M23-1520","article-title":"Gene therapy versus common care for eligible individuals with sickle cell disease in the United States: a cost-effectiveness analysis","volume":"177","author":"Basu","year":"2024","journal-title":"Ann Intern Med"},{"issue":"6","key":"2026081311214223200_bib15","doi-asserted-by":"crossref","first-page":"779","DOI":"10.7326\/M22-3272","article-title":"Distributional cost-effectiveness of equity-enhancing gene therapy in sickle cell disease in the United States","volume":"176","author":"Goshua","year":"2023","journal-title":"Ann Intern Med"},{"key":"2026081311214223200_bib16","unstructured":"Beaudoin\n              FTP\n            , ThokalaP, NikitinD, . Gene therapies for sickle cell disease: effectiveness and value. 2023. Final Evidence Report. Institute for Clinical and Economic Review. Accessed 26 May 2026. https:\/\/icer.org\/wp-content\/uploads\/2023\/08\/ICER_SCD_Final_Report_FOR_PUBLICATION_082123_12MonthCheckUp.pdf."},{"issue":"1","key":"2026081311214223200_bib17","doi-asserted-by":"crossref","first-page":"10838","DOI":"10.1038\/s41598-021-90405-1","article-title":"Cost-effectiveness of a hypothetical cell or gene therapy cure for sickle cell disease","volume":"11","author":"Salcedo","year":"2021","journal-title":"Sci Rep"},{"issue":"6","key":"2026081311214223200_bib18","doi-asserted-by":"crossref","first-page":"693","DOI":"10.1007\/s40273-024-01385-9","article-title":"Cost-effectiveness of lovotibeglogene autotemcel (lovo-cel) gene therapy for patients with sickle cell disease and recurrent vaso-occlusive events in the United States","volume":"42","author":"Herring","year":"2024","journal-title":"Pharmacoeconomics"},{"issue":"18","key":"2026081311214223200_bib19","doi-asserted-by":"crossref","first-page":"1649","DOI":"10.1056\/NEJMoa2309676","article-title":"Exagamglogene autotemcel for severe sickle cell disease","volume":"390","author":"Frangoul","year":"2024","journal-title":"N Engl J Med"},{"issue":"1","key":"2026081311214223200_bib20","doi-asserted-by":"crossref","first-page":"25","DOI":"10.7326\/M20-1392","article-title":"A health opportunity cost threshold for cost-effectiveness analysis in the United States","volume":"174","author":"Vanness","year":"2021","journal-title":"Ann Intern Med"},{"issue":"3","key":"2026081311214223200_bib21","doi-asserted-by":"crossref","first-page":"457","DOI":"10.1016\/j.jval.2025.10.007","article-title":"Estimating the willingness-to-pay per quality-adjusted life-year to aid health technology assessment in India","volume":"29","author":"Chugh","year":"2026","journal-title":"Value Health"},{"issue":"6","key":"2026081311214223200_bib22","doi-asserted-by":"crossref","first-page":"e833","DOI":"10.1016\/S2214-109X(23)00162-6","article-title":"Determining the efficiency path to universal health coverage: cost-effectiveness thresholds for 174 countries based on growth in life expectancy and health expenditures","volume":"11","author":"Pichon-Riviere","year":"2023","journal-title":"Lancet Glob Health"},{"issue":"1","key":"2026081311214223200_bib23","doi-asserted-by":"crossref","first-page":"10","DOI":"10.1016\/j.jval.2021.10.008","article-title":"Consolidated health economic evaluation reporting standards (CHEERS) 2022 explanation and elaboration: a report of the ISPOR CHEERS II good practices task force","volume":"25","author":"Husereau","year":"2022","journal-title":"Value Health"},{"issue":"10","key":"2026081311214223200_bib24","doi-asserted-by":"crossref","first-page":"1093","DOI":"10.1001\/jama.2016.12195","article-title":"Recommendations for conduct, methodological practices, and reporting of cost-effectiveness analyses: second panel on cost-effectiveness in health and medicine","volume":"316","author":"Sanders","year":"2016","journal-title":"JAMA"},{"key":"2026081311214223200_bib77","unstructured":"Optum.com\n          . Optum Research Data Assets. Accessed 26 May 2026. https:\/\/www.optum.com\/content\/dam\/optum\/resources\/productSheets\/5302_Data_Assets_Chart_Sheet_ISPOR.pdf."},{"issue":"6","key":"2026081311214223200_bib25","first-page":"325.e1","article-title":"Long-term survival after hematopoietic cell transplant for sickle cell disease compared to the United States population","volume":"28","author":"St Martin","year":"2022","journal-title":"Transpl Cell Ther"},{"issue":"6","key":"2026081311214223200_bib26","doi-asserted-by":"crossref","first-page":"1023","DOI":"10.1002\/ajh.27295","article-title":"Favorable outcome of non-myeloablative allogeneic transplantation in adult patients with severe sickle cell disease: a single center experience of 200 patients","volume":"99","author":"Damlaj","year":"2024","journal-title":"Am J Hematol"},{"issue":"6","key":"2026081311214223200_bib27","first-page":"616","article-title":"Treatment-sensitive and treatment-dependent chronic graft-versus-host disease yield superior failure-free and overall survival compared to treatment-resistant chronic graft-versus-host disease","volume":"30","author":"El Jurdi","year":"2024","journal-title":"Transpl Cell Ther"},{"issue":"12","key":"2026081311214223200_bib28","doi-asserted-by":"crossref","first-page":"2227","DOI":"10.1200\/JCO.22.01203","article-title":"Secondary neoplasms after hematopoietic cell transplant for sickle cell disease","volume":"41","author":"Eapen","year":"2023","journal-title":"J Clin Oncol"},{"issue":"11","key":"2026081311214223200_bib29","doi-asserted-by":"crossref","first-page":"e1915374","DOI":"10.1001\/jamanetworkopen.2019.15374","article-title":"Estimated life expectancy and income of patients with sickle cell disease compared with those without sickle cell disease","volume":"2","author":"Lubeck","year":"2019","journal-title":"JAMA Netw Open"},{"issue":"4","key":"2026081311214223200_bib30","doi-asserted-by":"crossref","first-page":"e001274","DOI":"10.1136\/bmjopen-2012-001274","article-title":"Patient self-assessment of hospital pain, mood and health-related quality of life in adults with sickle cell disease","volume":"2","author":"Anie","year":"2012","journal-title":"BMJ Open"},{"issue":"3","key":"2026081311214223200_bib31","doi-asserted-by":"crossref","first-page":"397","DOI":"10.1007\/s10198-019-01136-0","article-title":"Health state utilities associated with treatment for transfusion-dependent \u03b2-thalassemia","volume":"21","author":"Matza","year":"2020","journal-title":"Eur J Health Econ"},{"issue":"6","key":"2026081311214223200_bib32","first-page":"898","article-title":"Frequent and long-term follow-up of health-related quality of life following allogeneic haematopoietic stem cell transplantation","volume":"24","author":"Frodin","year":"2015","journal-title":"Eur J Cancer Care (Engl)"},{"issue":"10","key":"2026081311214223200_bib34","doi-asserted-by":"crossref","first-page":"3057","DOI":"10.1182\/blood.V95.10.3057","article-title":"Bone marrow transplantation versus periodic prophylactic blood transfusion in sickle cell patients at high risk of ischemic stroke: a decision analysis","volume":"95","author":"Nietert","year":"2000","journal-title":"Blood"},{"key":"2026081311214223200_bib80","unstructured":"Form 8-K\n          . Vertex Pharmaceuticals Incorporated. United States Securties and Exchange Commission. Dec 2023. Accessed 26 May 2026. https:\/\/www.sec.gov\/ix?doc=\/Archives\/edgar\/data\/0000875320\/000087532023000054\/vrtx-20231208.htm."},{"key":"2026081311214223200_bib78","unstructured":"Addendum\n              A\n            , AddendumB. Quarterly Addenda Updates. CMS. Accessed 26 May 2026. https:\/\/www.cms.gov\/Medicare\/Medicare-Fee-for-Service-Payment\/HospitalOutpatientPPS\/Addendum-A-and-Addendum-B-Updates."},{"issue":"7","key":"2026081311214223200_bib35","first-page":"366","article-title":"The cost of hematopoietic stem-cell transplantation in the United States","volume":"10","author":"Broder","year":"2017","journal-title":"Am Health Drug Benefits"},{"key":"2026081311214223200_bib79","unstructured":"2025 Drug Payment Limit Files. ASP Pricing Files. CMS. Accessed 26 May 2026. https:\/\/www.cms.gov\/medicare\/payment\/part-b-drugs\/asp-pricing-files."},{"issue":"2","key":"2026081311214223200_bib36","doi-asserted-by":"crossref","first-page":"e0229710","DOI":"10.1371\/journal.pone.0229710","article-title":"Improved health care utilization and costs in transplanted versus non-transplanted adults with sickle cell disease","volume":"15","author":"Saraf","year":"2020","journal-title":"PLoS One"},{"issue":"5","key":"2026081311214223200_bib37","doi-asserted-by":"crossref","first-page":"755","DOI":"10.1080\/03007995.2021.1893676","article-title":"Healthcare resource utilization and costs among patients with steroid-resistant chronic graft-versus-host disease in the United States: a retrospective claims database analysis","volume":"37","author":"Yu","year":"2021","journal-title":"Curr Med Res Opin"},{"issue":"1","key":"2026081311214223200_bib38","doi-asserted-by":"crossref","first-page":"169","DOI":"10.1007\/s10198-020-01249-x","article-title":"Real-world study of direct medical and indirect costs and time spent in healthcare in patients with chronic graft versus host disease","volume":"22","author":"Schain","year":"2021","journal-title":"Eur J Health Econ"},{"issue":"4","key":"2026081311214223200_bib39","first-page":"349","article-title":"International recommendations for screening and preventative practices for long-term survivors of transplantation and cellular therapy: a 2023 update","volume":"30","author":"Rotz","year":"2024","journal-title":"Transpl Cell Ther"},{"key":"2026081311214223200_bib40","unstructured":"U.S. Bureau of Economic Analysis\n          . Personal consumption expenditures: services: health care (chain-type price index) [DHLCRG3A086NBEA], retrieved from FRED, Federal Reserve Bank of St. Louis. 2026. Accessed 26 May 2026. https:\/\/fred.stlouisfed.org\/series\/DHLCRG3A086NBEA."},{"issue":"6","key":"2026081311214223200_bib41","doi-asserted-by":"crossref","first-page":"621","DOI":"10.1016\/j.mjafi.2023.09.002","article-title":"Hematopoietic cell transplantation landscape in India","volume":"79","author":"Nair","year":"2023","journal-title":"Med J Armed Forces India"},{"issue":"4","key":"2026081311214223200_bib42","doi-asserted-by":"crossref","first-page":"192","DOI":"10.1053\/j.seminhematol.2023.08.002","article-title":"Current challenges and new approaches to implementing optimal management of sickle cell disease in sub-Saharan Africa","volume":"60","author":"Ally","year":"2023","journal-title":"Semin Hematol"},{"issue":"10","key":"2026081311214223200_bib43","doi-asserted-by":"crossref","first-page":"2119","DOI":"10.1016\/j.bbmt.2018.04.005","article-title":"Cost effectiveness of hematopoietic stem cell transplantation compared with transfusion chelation for treatment of thalassemia major","volume":"24","author":"John","year":"2018","journal-title":"Biol Blood Marrow Transpl"},{"issue":"5","key":"2026081311214223200_bib44","doi-asserted-by":"crossref","first-page":"e017887","DOI":"10.1136\/bmjgh-2024-017887","article-title":"Socioeconomic health and impact of sickle cell disease and vaso-occlusive crises in India: results from B-VOCAL study","volume":"10","author":"Jain","year":"2025","journal-title":"BMJ Glob Health"},{"issue":"1","key":"2026081311214223200_bib45","doi-asserted-by":"crossref","first-page":"234","DOI":"10.1182\/hematology.2024000550","article-title":"Transfusions, disease-modifying treatments, and curative therapies for sickle cell anemia in Africa: where are we now?","volume":"2024","author":"Odame","year":"2024","journal-title":"Hematol Am Soc Hematol Educ Program"},{"issue":"1","key":"2026081311214223200_bib46","doi-asserted-by":"crossref","first-page":"2739","DOI":"10.1038\/s41598-024-53121-0","article-title":"The value-based price of transformative gene therapy for sickle cell disease: a modeling analysis","volume":"14","author":"Morgan","year":"2024","journal-title":"Sci Rep"},{"key":"2026081311214223200_bib47","unstructured":"Ministry of Statistics & Programme Implementation\n          . Consumer Price Index Numbers on Base 2012=100 for Rural, Urban and Combined for the Month of December 2017. 2018. Accessed 26 May 2026. https:\/\/www.pib.gov.in\/PressReleasePage.aspx?PRID=1516561&reg=3&lang=1."},{"key":"2026081311214223200_bib48","unstructured":"Ministry of Statistics & Programme Implementation\n          . Consumer Price Index Numbers on Base 2012=100 for Rural, Urban and Combined for the Month of December 2024. 2025. Accessed 26 May 2026. https:\/\/www.pib.gov.in\/PressReleseDetailm.aspx?PRID=2092477&reg=3&lang=2."},{"key":"2026081311214223200_bib49","unstructured":"Ministry of Statistics & Programme Implementation\n          . Consumer Price Index Numbers on Base 2012=100 for Rural, Urban and Combined for the Month of December, 2025. 2026. Accessed 26 May 2026. https:\/\/www.pib.gov.in\/PressReleasePage.aspx?PRID=2213736&reg=3&lang=1."},{"key":"2026081311214223200_bib50","unstructured":"World Bank Group\n          . Official Exchange Rate (LCU per US$, Period Average). Accessed 26 May 2026. https:\/\/data.worldbank.org\/indicator\/PA.NUS.FCRF."},{"issue":"2","key":"2026081311214223200_bib51","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1016\/j.jval.2022.07.020","article-title":"Gradient boosted tree approaches for mapping European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 Onto 5-Level Version of EQ-5D Index for patients with cancer","volume":"26","author":"Hagiwara","year":"2023","journal-title":"Value Health"},{"issue":"12","key":"2026081311214223200_bib52","doi-asserted-by":"crossref","first-page":"1162","DOI":"10.1097\/MLR.0b013e31814848f1","article-title":"US norms for six generic health-related quality-of-life indexes from the National Health Measurement study","volume":"45","author":"Fryback","year":"2007","journal-title":"Med Care"},{"issue":"24","key":"2026081311214223200_bib53","doi-asserted-by":"crossref","first-page":"6481","DOI":"10.1182\/bloodadvances.2025016701","article-title":"Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel","volume":"9","author":"Sharma","year":"2025","journal-title":"Blood Adv"},{"key":"2026081311214223200_bib54","doi-asserted-by":"crossref","first-page":"551170","DOI":"10.3389\/fped.2020.551170","article-title":"Hematopoietic cell transplantation for sickle cell disease","volume":"8","author":"Krishnamurti","year":"2021","journal-title":"Front Pediatr"},{"issue":"4","key":"2026081311214223200_bib55","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1016\/j.hemonc.2017.05.008","article-title":"Hematopoietic stem cell transplantation for sickle cell disease: the changing landscape","volume":"10","author":"Kassim","year":"2017","journal-title":"Hematol Oncol Stem Cell Ther"},{"issue":"18","key":"2026081311214223200_bib56","doi-asserted-by":"crossref","first-page":"3668","DOI":"10.1182\/bloodadvances.2021004394C","article-title":"American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation","volume":"5","author":"Kanter","year":"2021","journal-title":"Blood Adv"},{"issue":"17","key":"2026081311214223200_bib57","doi-asserted-by":"crossref","first-page":"4502","DOI":"10.1182\/bloodadvances.2025016053","article-title":"An ethical allocation scheme for scarce gene therapies in sickle cell disease and transfusion-dependent \u03b2-thalassemia","volume":"9","author":"Kelkar","year":"2025","journal-title":"Blood Adv"},{"issue":"5","key":"2026081311214223200_bib58","doi-asserted-by":"crossref","first-page":"2172","DOI":"10.1016\/j.ymthe.2025.03.016","article-title":"Current and future treatments for sickle cell disease: from hematopoietic stem cell transplantation to in vivo gene therapy","volume":"33","author":"Ball","year":"2025","journal-title":"Mol Ther"},{"issue":"1","key":"2026081311214223200_bib59","doi-asserted-by":"crossref","first-page":"6291","DOI":"10.1038\/s41467-023-41153-5","article-title":"Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model","volume":"14","author":"Uchida","year":"2023","journal-title":"Nat Commun"},{"issue":"2","key":"2026081311214223200_bib60","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1016\/j.jval.2024.10.3848","article-title":"Financial impacts of paying for gene therapy for sickle cell disease under alternative pricing and financing mechanisms","volume":"28","author":"Basu","year":"2025","journal-title":"Value Health"},{"issue":"4","key":"2026081311214223200_bib61","doi-asserted-by":"crossref","first-page":"1185","DOI":"10.1007\/s11606-025-09865-0","article-title":"Modeling the budgetary impacts of sickle cell disease gene therapies on state Medicaid programs","volume":"41","author":"Harvey","year":"2025","journal-title":"J Gen Intern Med"},{"issue":"12","key":"2026081311214223200_bib62","doi-asserted-by":"crossref","first-page":"e14462","DOI":"10.2196\/14462","article-title":"Comparative effectiveness of a web-based patient decision aid for therapeutic options for sickle cell disease: randomized controlled trial","volume":"21","author":"Krishnamurti","year":"2019","journal-title":"J Med Internet Res"},{"issue":"5","key":"2026081311214223200_bib63","doi-asserted-by":"crossref","first-page":"1041","DOI":"10.1016\/j.bbmt.2017.11.018","article-title":"Experiences and decision making in hematopoietic stem cell transplant in sickle cell disease: patients' and caregivers' perspectives","volume":"24","author":"Khemani","year":"2018","journal-title":"Biol Blood Marrow Transpl"},{"issue":"1","key":"2026081311214223200_bib64","doi-asserted-by":"crossref","first-page":"e28749","DOI":"10.1002\/pbc.28749","article-title":"Primary caregiver decision-making in hematopoietic cell transplantation and gene therapy for sickle cell disease","volume":"68","author":"Sinha","year":"2021","journal-title":"Pediatr Blood Cancer"},{"issue":"8","key":"2026081311214223200_bib65","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1038\/sj.bmt.1705622","article-title":"A survey on patient perception of reduced-intensity transplantation in adults with sickle cell disease","volume":"39","author":"Chakrabarti","year":"2007","journal-title":"Bone Marrow Transpl"},{"issue":"11","key":"2026081311214223200_bib66","doi-asserted-by":"crossref","first-page":"1416","DOI":"10.1038\/bmt.2016.164","article-title":"A scoping review of caregiver burden during allogeneic HSCT: lessons learned and future directions","volume":"51","author":"Applebaum","year":"2016","journal-title":"Bone Marrow Transpl"},{"issue":"4","key":"2026081311214223200_bib67","doi-asserted-by":"crossref","first-page":"289","DOI":"10.1097\/MPH.0b013e31828d5e6a","article-title":"Barriers to hematopoietic cell transplantation clinical trial participation of African American and Black youth with sickle cell disease and their parents","volume":"35","author":"Omondi","year":"2013","journal-title":"J Pediatr Hematol Oncol"},{"issue":"1","key":"2026081311214223200_bib68","doi-asserted-by":"crossref","first-page":"S78","DOI":"10.1016\/j.amepre.2016.01.024","article-title":"Mistrust of pediatric sickle cell disease clinical trials research","volume":"51","author":"Stevens","year":"2016","journal-title":"Am J Prev Med"},{"issue":"suppl 1","key":"2026081311214223200_bib69","first-page":"602","article-title":"Edwards JG, Dosunmu-Ogundi A, Oni MO, et al. Opportunities for standardizing conversations about curative therapy for sickle cell disease [abstract]","volume":"144","year":"2024","journal-title":"Blood"},{"issue":"11","key":"2026081311214223200_bib70","doi-asserted-by":"crossref","first-page":"e71464","DOI":"10.1002\/hsr2.71464","article-title":"Advancing hematopoietic stem cell transplantation (HSCT) in Africa: a pathway to curing hematological disorders","volume":"8","author":"Ogunniyi","year":"2025","journal-title":"Health Sci Rep"},{"issue":"3-4","key":"2026081311214223200_bib71","doi-asserted-by":"crossref","first-page":"216","DOI":"10.1038\/s41434-021-00284-4","article-title":"Towards access for all: 1st working group report for the Global Gene Therapy Initiative (GGTI)","volume":"30","author":"Adair","year":"2023","journal-title":"Gene Ther"},{"issue":"11","key":"2026081311214223200_bib72","doi-asserted-by":"crossref","first-page":"3101","DOI":"10.1016\/j.ymthe.2021.05.024","article-title":"Collaborative science to advance gene therapies in resource-limited parts of the world","volume":"29","author":"McCune","year":"2021","journal-title":"Mol Ther"},{"issue":"12","key":"2026081311214223200_bib73","doi-asserted-by":"crossref","first-page":"2451","DOI":"10.1038\/s41591-022-02033-5","article-title":"Africa must participate in finding a gene therapy cure for sickle-cell disease","volume":"28","author":"Moshi","year":"2022","journal-title":"Nat Med"},{"key":"2026081311214223200_bib74","unstructured":"Lakshman\n              A\n            \n          . India getting close to developing gene therapy for sickle cell disease. The Hindu. 20 June 2024. Accessed 26 May 2026. https:\/\/www.thehindu.com\/sci-tech\/science\/india-getting-close-to-developing-gene-therapy-for-sickle-cell-disease-say-officials\/article68308487.ece."},{"key":"2026081311214223200_bib75","unstructured":"Novartis\n          . Novartis and the Bill & Melinda Gates Foundation Collaborate to Discover and Develop an Accessible in vivo Gene Therapy for Sickle Cell Disease. Accessed 26 May 2026. https:\/\/www.novartis.com\/news\/media-releases\/novartis-and-bill-melinda-gates-foundation-collaborate-discover-and-develop-accessible-vivo-gene-therapy-sickle-cell-disease."},{"key":"2026081311214223200_bib76","unstructured":"Cell and Gene Therapy (CGT) Access Model. 2024. U.S. Centers for Medicare & Medicaid Services. Accessed 26 May 2026. https:\/\/www.cms.gov\/priorities\/innovation\/innovation-models\/cgt."}],"container-title":["Blood"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/ashpublications.org\/blood\/article-pdf\/148\/7\/896\/2537523\/blood_bld-2025-032290-main.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/ashpublications.org\/blood\/article-pdf\/148\/7\/896\/2537523\/blood_bld-2025-032290-main.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,13]],"date-time":"2026-08-13T15:21:59Z","timestamp":1786634519000},"score":1,"resource":{"primary":{"URL":"https:\/\/ashpublications.org\/blood\/article\/148\/7\/896\/568518\/Haploidentical-transplant-gene-therapy-and"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,8,13]]},"references-count":79,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2026,8,13]]}},"URL":"https:\/\/doi.org\/10.1182\/blood.2025032290","relation":{},"ISSN":["0006-4971","1528-0020"],"issn-type":[{"value":"0006-4971","type":"print"},{"value":"1528-0020","type":"electronic"}],"subject":[],"published-other":{"date-parts":[[2026,8,13]]},"published":{"date-parts":[[2026,8,13]]}}}