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  • Schoenmakers, Daphne H.Vrije Universiteit Amsterdam,Amsterdam Neuroscience,University of Amsterdam (author)

Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries

  • Article/chapterEnglish2024

Publisher, publication year, extent ...

  • 2024

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  • LIBRIS-ID:oai:lup.lub.lu.se:f45eb2ee-74e1-4296-b42d-e32ef49c7a93
  • https://lup.lub.lu.se/record/f45eb2ee-74e1-4296-b42d-e32ef49c7a93URI
  • https://doi.org/10.1186/s13023-024-03075-3DOI

Supplementary language notes

  • Language:English
  • Summary in:English

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  • Subject category:art swepub-publicationtype
  • Subject category:ref swepub-contenttype

Notes

  • Background: For decades, early allogeneic stem cell transplantation (HSCT) has been used to slow neurological decline in metachromatic leukodystrophy (MLD). There is lack of consensus regarding who may benefit, and guidelines are lacking. Clinical practice relies on limited literature and expert opinions. The European Reference Network for Rare Neurological Diseases (ERN-RND) and the MLD initiative facilitate expert panels for treatment advice, but some countries are underrepresented. This study explores organizational and clinical HSCT practices for MLD in Europe and neighboring countries to enhance optimization and harmonization of cross-border MLD care. Methods: A web-based EUSurvey was distributed through the ERN-RND and the European Society for Blood and Marrow Transplantation Inborn Errors Working Party. Personal invitations were sent to 89 physicians (43 countries) with neurological/metabolic/hematological expertise. The results were analyzed and visualized using Microsoft Excel and IBM SPSS statistics. Results: Of the 30 countries represented by 42 respondents, 23 countries offer HSCT for MLD. The treatment is usually available in 1–3 centers per country (18/23, 78%). Most countries have no or very few MLD patients transplanted during the past 1–5 years. The eligibility criteria regarding MLD subtype, motor function, IQ, and MRI largely differ across countries. Conclusion: HSCT for MLD is available in most European countries, but uncertainties exist in Eastern and South-Eastern Europe. Applied eligibility criteria and management vary and may not align with the latest scientific insights, indicating physicians’ struggle in providing evidence-based care. Interaction between local physicians and international experts is crucial for adequate treatment decision-making and cross-border care in the rapidly changing MLD field.

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Added entries (persons, corporate bodies, meetings, titles ...)

  • Mochel, FannyBrain and Spine Institute (ICM) (author)
  • Adang, Laura A.The Children's Hospital of Philadelphia (author)
  • Boelens, Jaap JanMemorial Sloan-Kettering Cancer Center (author)
  • Calbi, ValeriaSan Raffaele Hospital (author)
  • Eklund, Erik A.Lund University,Lunds universitet,Pediatrik, Lund,Sektion V,Institutionen för kliniska vetenskaper, Lund,Medicinska fakulteten,Paediatrics (Lund),Section V,Department of Clinical Sciences, Lund,Faculty of Medicine,Skåne University Hospital(Swepub:lu)medk-eek (author)
  • Grønborg, Sabine W.Copenhagen University Hospital (author)
  • Fumagalli, Francesca (author)
  • Groeschel, SamuelUniversity Hospital of Tubingen (author)
  • Lindemans, CarolineWilhelmina Children’s Hospital,Princess Maxima Center for Pediatric Oncology/Hematology (author)
  • Sevin, CarolineAssistance Publique des Hôpitaux de Paris (author)
  • Schöls, LudgerUniversity of Tübingen,German Center for Neurodegenerative Diseases (DZNE), Bonn (author)
  • Ram, DipakRoyal Manchester Children's Hospital (author)
  • Zerem, AyeletTel-Aviv University (author)
  • Graessner, HolmUniversity of Tübingen (author)
  • Wolf, Nicole I.Amsterdam Neuroscience,Vrije Universiteit Amsterdam (author)
  • Vrije Universiteit AmsterdamAmsterdam Neuroscience (creator_code:org_t)

Related titles

  • In:Orphanet Journal of Rare Diseases19:11750-1172

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