{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,3]],"date-time":"2025-10-03T18:05:36Z","timestamp":1759514736258,"version":"3.37.3"},"reference-count":50,"publisher":"Springer Science and Business Media LLC","issue":"7","license":[{"start":{"date-parts":[[2024,4,2]],"date-time":"2024-04-02T00:00:00Z","timestamp":1712016000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2024,4,2]],"date-time":"2024-04-02T00:00:00Z","timestamp":1712016000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/100016062","name":"Stichting Metakids","doi-asserted-by":"publisher","award":["2017-073"],"award-info":[{"award-number":["2017-073"]}],"id":[{"id":"10.13039\/100016062","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J Neurol"],"published-print":{"date-parts":[[2024,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Metachromatic leukodystrophy (MLD) is a neuro-metabolic disorder due to arylsulfatase A deficiency, causing demyelination of the central and peripheral nervous system. Hematopoietic cell transplantation (HCT) can provide a symptomatic and survival benefit for pre-symptomatic and early symptomatic patients by stabilizing CNS disease. This case series, however, illustrates the occurrence of severely progressive polyneuropathy shortly after HCT in two patients with late-infantile, one with late-juvenile, and one with adult MLD, leading to the inability to walk or sit without support. The patients had demyelinating polyneuropathy before HCT, performed at the ages of 2\u00a0years in the first two patients and at 14 and 23\u00a0years in the other two patients. The myeloablative conditioning regimen consisted of busulfan, fludarabine and, in one case, rituximab, with anti-thymocyte globulin, cyclosporine, steroids, and\/or mycophenolate mofetil for GvHD prophylaxis. Polyneuropathy after HCT progressed parallel with tapering immunosuppression and paralleled bouts of infection and graft-versus-host disease (GvHD). Differential diagnoses included MLD progression, neurological GvHD or another (auto)inflammatory cause. Laboratory, electroneurography and pathology investigations were inconclusive. In two patients, treatment with immunomodulatory drugs led to temporary improvement, but not sustained stabilization of polyneuropathy. One patient showed recovery to pre-HCT functioning, except for a Holmes-like tremor, for which a peripheral origin cannot be excluded. One patient showed marginal response to immunosuppressive treatment and died ten months after HCT due to respiratory failure. The extensive diagnostic and therapeutic attempts highlight the challenge of characterizing and treating progressive polyneuropathy in patients with MLD shortly after HCT. We advise to consider repeat electro-neurography and possibly peripheral nerve biopsy in such patients. Nerve conduction blocks, evidence of the presence of T lymphocytes and macrophages in the neuronal and surrounding nerve tissue, and beneficial effects of immunomodulatory drugs may indicate a partially (auto)immune-mediated pathology. Polyneuropathy may cause major residual disease burden after HCT. MLD patients with progressive polyneuropathy could potentially benefit from a more intensified immunomodulatory drug regime following HCT, especially at times of immune activation.<\/jats:p>","DOI":"10.1007\/s00415-024-12322-3","type":"journal-article","created":{"date-parts":[[2024,4,2]],"date-time":"2024-04-02T12:01:47Z","timestamp":1712059307000},"page":"4028-4038","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Progressive demyelinating polyneuropathy after hematopoietic cell transplantation in metachromatic leukodystrophy: a case series"],"prefix":"10.1007","volume":"271","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2945-6784","authenticated-orcid":false,"given":"Shanice","family":"Beerepoot","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2232-6952","authenticated-orcid":false,"given":"Jaap Jan","family":"Boelens","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3984-5620","authenticated-orcid":false,"given":"Caroline","family":"Lindemans","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7470-2994","authenticated-orcid":false,"given":"Moniek A.","family":"de Witte","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3406-817X","authenticated-orcid":false,"given":"Stefan","family":"Nierkens","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4649-6534","authenticated-orcid":false,"given":"Alexander F. J. E.","family":"Vrancken","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8912-0954","authenticated-orcid":false,"given":"Marjo S.","family":"van der Knaap","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7689-3042","authenticated-orcid":false,"given":"Marianna","family":"Bugiani","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1721-0728","authenticated-orcid":false,"given":"Nicole I.","family":"Wolf","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2024,4,2]]},"reference":[{"issue":"11","key":"12322_CR1","doi-asserted-by":"crossref","first-page":"805","DOI":"10.1111\/j.1471-4159.1963.tb11905.x","volume":"10","author":"JH Austin","year":"1963","unstructured":"Austin JH et al (1963) A controlled study of enzymic activities in three human disorders of glycolipid metabolism. J Neurochem 10(11):805\u2013816","journal-title":"J Neurochem"},{"key":"12322_CR2","first-page":"3695","volume-title":"The metabolic and molecular bases of inherited disease","author":"K Von Figura","year":"2001","unstructured":"Von Figura K, Gieselmann V, Jaeken J (2001) Metachromatic leukodystrophy. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds) The metabolic and molecular bases of inherited disease. McGraw-Hill, New York, pp 3695\u20133724"},{"issue":"12","key":"12322_CR3","doi-asserted-by":"crossref","first-page":"1698","DOI":"10.1136\/jnnp.2005.063776","volume":"76","author":"PS Bindu","year":"2005","unstructured":"Bindu PS et al (2005) Peripheral neuropathy in metachromatic leucodystrophy. A study of 40 cases from south India. J Neurol Neurosurg Psychiatry 76(12):1698\u2013701","journal-title":"J Neurol Neurosurg Psychiatry"},{"issue":"3","key":"12322_CR4","doi-asserted-by":"crossref","first-page":"659","DOI":"10.1007\/s00415-018-8765-3","volume":"265","author":"DF van Rappard","year":"2018","unstructured":"van Rappard DF et al (2018) Diffusion tensor imaging in metachromatic leukodystrophy. J Neurol 265(3):659\u2013668","journal-title":"J Neurol"},{"issue":"4","key":"12322_CR5","doi-asserted-by":"crossref","first-page":"616","DOI":"10.1016\/j.bbmt.2013.01.010","volume":"19","author":"HR Martin","year":"2013","unstructured":"Martin HR et al (2013) Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic leukodystrophy. Biol Blood Marrow Transplant 19(4):616\u2013624","journal-title":"Biol Blood Marrow Transplant"},{"key":"12322_CR6","first-page":"681","volume-title":"Atlas of metabolic disease","author":"WN Nyhan","year":"2005","unstructured":"Nyhan WN, Barshop BA, Ozand PT (2005) Metachromatic leukodystrophy. In: Nyhan WN, Barshop BA, Ozand PT (eds) Atlas of metabolic disease. Hodder Arnold, London, pp 681\u2013685"},{"issue":"4","key":"12322_CR7","doi-asserted-by":"crossref","first-page":"338","DOI":"10.1055\/s-0028-1091493","volume":"9","author":"JA Luijten","year":"1978","unstructured":"Luijten JA et al (1978) Metachromatic leukodystrophy: a comparative study of the ultrastructural findings in the peripheral nervous system of three cases, one of the late infantile, one of the juvenile and one of the adult form of the disease. Neuropadiatrie 9(4):338\u2013350","journal-title":"Neuropadiatrie"},{"issue":"2","key":"12322_CR8","doi-asserted-by":"crossref","first-page":"261","DOI":"10.1016\/j.beem.2014.10.001","volume":"29","author":"DF van Rappard","year":"2015","unstructured":"van Rappard DF, Boelens JJ, Wolf NI (2015) Metachromatic leukodystrophy: disease spectrum and approaches for treatment. Best Pract Res Clin Endocrinol Metab 29(2):261\u2013273","journal-title":"Best Pract Res Clin Endocrinol Metab"},{"issue":"1","key":"12322_CR9","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1002\/humu.22919","volume":"37","author":"M Cesani","year":"2016","unstructured":"Cesani M et al (2016) Mutation update of ARSA and PSAP genes causing metachromatic leukodystrophy. Hum Mutat 37(1):16\u201327","journal-title":"Hum Mutat"},{"issue":"24","key":"12322_CR10","doi-asserted-by":"crossref","first-page":"3098","DOI":"10.1182\/blood-2016-03-708479","volume":"127","author":"DF van Rappard","year":"2016","unstructured":"van Rappard DF et al (2016) Efficacy of hematopoietic cell transplantation in metachromatic leukodystrophy: the Dutch experience. Blood 127(24):3098\u20133101","journal-title":"Blood"},{"issue":"9","key":"12322_CR11","doi-asserted-by":"crossref","first-page":"1133","DOI":"10.1001\/jamaneurol.2016.2067","volume":"73","author":"S Groeschel","year":"2016","unstructured":"Groeschel S et al (2016) Long-term outcome of allogeneic hematopoietic stem cell transplantation in patients with juvenile metachromatic leukodystrophy compared with nontransplanted control patients. JAMA Neurol 73(9):1133\u20131140","journal-title":"JAMA Neurol"},{"issue":"1","key":"12322_CR12","doi-asserted-by":"crossref","first-page":"240","DOI":"10.1186\/s13023-019-1220-4","volume":"14","author":"S Beerepoot","year":"2019","unstructured":"Beerepoot S et al (2019) Peripheral neuropathy in metachromatic leukodystrophy: current status and future perspective. Orphanet J Rare Dis 14(1):240","journal-title":"Orphanet J Rare Dis"},{"issue":"11","key":"12322_CR13","doi-asserted-by":"crossref","first-page":"1439","DOI":"10.1016\/j.bbmt.2009.07.013","volume":"15","author":"P Barba","year":"2009","unstructured":"Barba P et al (2009) Early and late neurological complications after reduced-intensity conditioning allogeneic stem cell transplantation. Biol Blood Marrow Transplant 15(11):1439\u20131446","journal-title":"Biol Blood Marrow Transplant"},{"issue":"6","key":"12322_CR14","doi-asserted-by":"crossref","first-page":"616","DOI":"10.1016\/j.pediatrneurol.2014.01.028","volume":"50","author":"R Dubey","year":"2014","unstructured":"Dubey R et al (2014) Leukodystrophy presenting as acute-onset polyradiculoneuropathy. Pediatr Neurol 50(6):616\u2013618","journal-title":"Pediatr Neurol"},{"issue":"2","key":"12322_CR15","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1007\/s11060-012-0962-9","volume":"110","author":"AM Delios","year":"2012","unstructured":"Delios AM, Rosenblum M, Jakubowski AA, DeAngelis LM (2012) Central and peripheral nervous system immune mediated demyelinating disease after allogeneic hemopoietic stem cell transplantation for hematologic disease. J Neurooncol 110(2):251\u2013256","journal-title":"J Neurooncol"},{"issue":"10","key":"12322_CR16","doi-asserted-by":"crossref","first-page":"2852","DOI":"10.1093\/brain\/awq245","volume":"133","author":"O Grauer","year":"2010","unstructured":"Grauer O et al (2010) Neurological manifestations of chronic graft-versus-host disease after allogeneic haematopoietic stem cell transplantation: report from the Consensus Conference on Clinical Practice in chronic graft-versus-host disease. Brain 133(10):2852\u201365","journal-title":"Brain"},{"issue":"4","key":"12322_CR17","doi-asserted-by":"crossref","first-page":"289","DOI":"10.1007\/s10048-020-00621-6","volume":"21","author":"S Beerepoot","year":"2020","unstructured":"Beerepoot S et al (2020) Metachromatic leukodystrophy genotypes in The Netherlands reveal novel pathogenic ARSA variants in non-Caucasian patients. Neurogenetics 21(4):289\u2013299","journal-title":"Neurogenetics"},{"issue":"2","key":"12322_CR18","doi-asserted-by":"crossref","first-page":"169","DOI":"10.1002\/acn3.50975","volume":"7","author":"NI Wolf","year":"2020","unstructured":"Wolf NI et al (2020) Metachromatic leukodystrophy and transplantation: remyelination, no cross-correction. Ann Clin Transl Neurol 7(2):169\u2013180","journal-title":"Ann Clin Transl Neurol"},{"key":"12322_CR19","doi-asserted-by":"crossref","first-page":"300","DOI":"10.1136\/bmj.4.5626.300","volume":"4","author":"H Aziz","year":"1968","unstructured":"Aziz H, Pearce J (1968) Peripheral neuropathy in metachromatic leukodystrophy. BMJ 4:300","journal-title":"BMJ"},{"issue":"1","key":"12322_CR20","doi-asserted-by":"crossref","first-page":"30","DOI":"10.1136\/jnnp.36.1.30","volume":"36","author":"KL De Silva","year":"1973","unstructured":"De Silva KL, Pearce J (1973) Neuropathy of metachromatic leucodystrophy. J Neurol Neurosurg Psychiatry 36(1):30\u201333","journal-title":"J Neurol Neurosurg Psychiatry"},{"issue":"5","key":"12322_CR21","doi-asserted-by":"crossref","first-page":"318","DOI":"10.1055\/s-0039-1692646","volume":"50","author":"P Madaan","year":"2019","unstructured":"Madaan P et al (2019) Saposin B-deficient metachromatic leukodystrophy mimicking acute flaccid paralysis. Neuropediatrics 50(5):318\u2013321","journal-title":"Neuropediatrics"},{"key":"12322_CR22","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1016\/j.ejpn.2022.01.020","volume":"37","author":"S Beerepoot","year":"2022","unstructured":"Beerepoot S et al (2022) Acute-onset paralytic strabismus in toddlers is important to consider as a potential early sign of late-infantile Metachromatic Leukodystrophy. Eur J Paediatr Neurol 37:87\u201393","journal-title":"Eur J Paediatr Neurol"},{"issue":"6","key":"12322_CR23","doi-asserted-by":"crossref","first-page":"291","DOI":"10.1055\/s-0030-1249654","volume":"40","author":"G Morana","year":"2009","unstructured":"Morana G et al (2009) Enhancing cranial nerves and cauda equina: an emerging magnetic resonance imaging pattern in metachromatic leukodystrophy and krabbe disease. Neuropediatrics 40(6):291\u2013294","journal-title":"Neuropediatrics"},{"issue":"5","key":"12322_CR24","doi-asserted-by":"crossref","first-page":"E41","DOI":"10.1002\/mus.25737","volume":"56","author":"HD Gonorazky","year":"2017","unstructured":"Gonorazky HD et al (2017) Subacute demyelinating peripheral neuropathy as a novel presentation of late infantile metachromatic leukodystrophy. Muscle Nerve 56(5):E41\u2013E44","journal-title":"Muscle Nerve"},{"issue":"3","key":"12322_CR25","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1016\/j.ejpn.2008.04.011","volume":"13","author":"E Haberlandt","year":"2009","unstructured":"Haberlandt E et al (2009) Peripheral neuropathy as the sole initial finding in three children with infantile metachromatic leukodystrophy. Eur J Paediatr Neurol 13(3):257\u2013260","journal-title":"Eur J Paediatr Neurol"},{"issue":"3","key":"12322_CR26","doi-asserted-by":"crossref","first-page":"237","DOI":"10.1016\/S0887-8994(96)00164-6","volume":"15","author":"Y Nevo","year":"1996","unstructured":"Nevo Y, Pestronk A, Lopate G, Carroll SL (1996) Neuropathy of metachromatic leukodystrophy: improvement with immunomodulation. Pediatr Neurol 15(3):237\u2013239","journal-title":"Pediatr Neurol"},{"issue":"2","key":"12322_CR27","doi-asserted-by":"crossref","first-page":"134","DOI":"10.1177\/1971400916633479","volume":"29","author":"D Roi","year":"2016","unstructured":"Roi D et al (2016) Thickening of the optic nerves in metachromatic leucodystrophy: a new MRI finding. Neuroradiol J 29(2):134\u2013136","journal-title":"Neuroradiol J"},{"issue":"2","key":"12322_CR28","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1212\/WNL.17.2.103","volume":"17","author":"A Yudell","year":"1967","unstructured":"Yudell A, Gomez MR, Lambert EH, Dockerty MB (1967) The neuropathy of sulfatide lipidosis (metachromatic leukodystrophy). Neurology 17(2):103\u201311 (passim)","journal-title":"Neurology"},{"issue":"5","key":"12322_CR29","doi-asserted-by":"crossref","first-page":"383","DOI":"10.1017\/S001216220600082X","volume":"48","author":"B Anlar","year":"2006","unstructured":"Anlar B, Waye JS, Eng B, Oguz KK (2006) Atypical clinical course in juvenile metachromatic leukodystrophy involving novel arylsulfatase A gene mutations. Dev Med Child Neurol 48(5):383\u2013387","journal-title":"Dev Med Child Neurol"},{"issue":"797","key":"12322_CR30","doi-asserted-by":"crossref","first-page":"192","DOI":"10.1136\/pgmj.68.797.192","volume":"68","author":"M Sadeh","year":"1992","unstructured":"Sadeh M, Kuritzky A, Ben-David E, Goldhammer Y (1992) Adult metachromatic leukodystrophy with an unusual relapsing-remitting course. Postgrad Med J 68(797):192\u2013195","journal-title":"Postgrad Med J"},{"issue":"1","key":"12322_CR31","doi-asserted-by":"crossref","DOI":"10.1212\/NXI.0000000000000922","volume":"8","author":"K Meier","year":"2021","unstructured":"Meier K, G\u00e4rtner J, Huppke P (2021) Tumefactive inflammatory lesions in juvenile metachromatic leukodystrophy. Neurol Neuroimmunol Neuroinflamm 8(1):e922","journal-title":"Neurol Neuroimmunol Neuroinflamm"},{"issue":"4","key":"12322_CR32","doi-asserted-by":"crossref","first-page":"141","DOI":"10.7224\/1537-2073-8.4.141","volume":"8","author":"M Kaufman","year":"2006","unstructured":"Kaufman M (2006) Juvenile metachromatic leukodystrophy mimicking relapsing-remitting multiple sclerosis. Int J MS Care 8(4):141\u2013143","journal-title":"Int J MS Care"},{"key":"12322_CR33","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000201230","author":"G Olive-Cirera","year":"2022","unstructured":"Olive-Cirera G, Martinez-Gonzalez MJ, Armangue T (2022) Pearls & oy-sters: tumefactive demyelinating lesions with MOG antibodies preceding late-infantile metachromatic leukodystrophy. Neurology. https:\/\/doi.org\/10.1212\/WNL.0000000000201230","journal-title":"Neurology"},{"issue":"6","key":"12322_CR34","doi-asserted-by":"crossref","first-page":"945","DOI":"10.4103\/1673-5374.233431","volume":"13","author":"AO Dulamea","year":"2018","unstructured":"Dulamea AO, Lupescu IG (2018) Neurological complications of hematopoietic cell transplantation in children and adults. Neural Regen Res 13(6):945\u2013954","journal-title":"Neural Regen Res"},{"issue":"4","key":"12322_CR35","doi-asserted-by":"crossref","first-page":"480","DOI":"10.1002\/mus.24724","volume":"52","author":"KM Ruzhansky","year":"2015","unstructured":"Ruzhansky KM, Brannagan TH 3rd (2015) Neuromuscular complications of hematopoietic stem cell transplantation. Muscle Nerve 52(4):480\u2013487","journal-title":"Muscle Nerve"},{"issue":"6","key":"12322_CR36","doi-asserted-by":"crossref","first-page":"638","DOI":"10.1136\/jnnp-2013-306657","volume":"85","author":"C Karam","year":"2014","unstructured":"Karam C et al (2014) Immune-mediated neuropathies following stem cell transplantation. J Neurol Neurosurg Psychiatry 85(6):638\u2013642","journal-title":"J Neurol Neurosurg Psychiatry"},{"issue":"1\u20132","key":"12322_CR37","doi-asserted-by":"crossref","first-page":"114","DOI":"10.1016\/j.jns.2010.01.020","volume":"292","author":"J Thone","year":"2010","unstructured":"Thone J et al (2010) Guillain-Barre syndrome as leading manifestation of graft-versus-host disease in an allogeneic bone marrow transplanted patient. J Neurol Sci 292(1\u20132):114\u2013116","journal-title":"J Neurol Sci"},{"key":"12322_CR38","doi-asserted-by":"crossref","first-page":"52","DOI":"10.1016\/j.ensci.2016.08.001","volume":"4","author":"T Yoshida","year":"2016","unstructured":"Yoshida T et al (2016) Guillain-Barre syndrome after allogeneic bone marrow transplantation: case report and literature review. eNeurologicalSci 4:52\u201355","journal-title":"eNeurologicalSci"},{"issue":"6","key":"12322_CR39","doi-asserted-by":"crossref","first-page":"1711","DOI":"10.1212\/WNL.49.6.1711","volume":"49","author":"PY Wen","year":"1997","unstructured":"Wen PY et al (1997) Guillain-Barre syndrome following allogeneic bone marrow transplantation. Neurology 49(6):1711\u20131714","journal-title":"Neurology"},{"issue":"9","key":"12322_CR40","doi-asserted-by":"crossref","first-page":"2337","DOI":"10.1007\/s00520-014-2225-0","volume":"22","author":"S Koeppen","year":"2014","unstructured":"Koeppen S, Thirugnanasambanthan A, Koldehoff M (2014) Neuromuscular complications after hematopoietic stem cell transplantation. Support Care Cancer 22(9):2337\u20132341","journal-title":"Support Care Cancer"},{"issue":"1","key":"12322_CR41","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1046\/j.0919-6544.2002.00419_22_1.x","volume":"22","author":"T Nagashima","year":"2002","unstructured":"Nagashima T et al (2002) Chronic demyelinating polyneuropathy in graft-versus-host disease following allogeneic bone marrow transplantation. Neuropathology 22(1):1\u20138","journal-title":"Neuropathology"},{"issue":"6","key":"12322_CR42","doi-asserted-by":"crossref","first-page":"617","DOI":"10.1038\/sj.bmt.1705283","volume":"37","author":"G Fujisaki","year":"2006","unstructured":"Fujisaki G et al (2006) Guillain-Barre syndrome associated with rapid immune reconstitution following allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplant 37(6):617\u2013619","journal-title":"Bone Marrow Transplant"},{"issue":"2","key":"12322_CR43","doi-asserted-by":"crossref","first-page":"137","DOI":"10.1038\/sj.bmt.1704538","volume":"34","author":"I Avivi","year":"2004","unstructured":"Avivi I et al (2004) Neurological complications following alemtuzumab-based reduced-intensity allogeneic transplantation. Bone Marrow Transplant 34(2):137\u2013142","journal-title":"Bone Marrow Transplant"},{"issue":"8","key":"12322_CR44","doi-asserted-by":"crossref","first-page":"1169","DOI":"10.1097\/00007890-200104270-00026","volume":"71","author":"KR Bulsara","year":"2001","unstructured":"Bulsara KR et al (2001) Guillain-Barre syndrome in organ and bone marrow transplant patients. Transplantation 71(8):1169\u20131172","journal-title":"Transplantation"},{"issue":"10043","key":"12322_CR45","doi-asserted-by":"crossref","first-page":"476","DOI":"10.1016\/S0140-6736(16)30374-9","volume":"388","author":"M Sessa","year":"2016","unstructured":"Sessa M et al (2016) Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1\/2 trial. Lancet 388(10043):476\u2013487","journal-title":"Lancet"},{"issue":"19","key":"12322_CR46","doi-asserted-by":"crossref","first-page":"10835","DOI":"10.3390\/ijms221910835","volume":"22","author":"P Baum","year":"2021","unstructured":"Baum P et al (2021) Inflammatory mechanisms in the pathophysiology of diabetic peripheral neuropathy (DN)-new aspects. Int J Mol Sci 22(19):10835","journal-title":"Int J Mol Sci"},{"key":"12322_CR47","doi-asserted-by":"publisher","DOI":"10.1111\/jns.12502","author":"MC Broers","year":"2022","unstructured":"Broers MC et al (2022) Epidemiology of chronic inflammatory demyelinating polyradiculoneuropathy in The Netherlands. J Peripher Nerv Syst. https:\/\/doi.org\/10.1111\/jns.12502","journal-title":"J Peripher Nerv Syst"},{"issue":"5","key":"12322_CR48","doi-asserted-by":"crossref","first-page":"1151","DOI":"10.1002\/jimd.12388","volume":"44","author":"F Fumagalli","year":"2021","unstructured":"Fumagalli F et al (2021) Metachromatic leukodystrophy: a single-center longitudinal study of 45 patients. J Inherit Metab Dis 44(5):1151\u20131164","journal-title":"J Inherit Metab Dis"},{"issue":"10322","key":"12322_CR49","doi-asserted-by":"crossref","first-page":"372","DOI":"10.1016\/S0140-6736(21)02017-1","volume":"399","author":"F Fumagalli","year":"2022","unstructured":"Fumagalli F et al (2022) Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1\/2 trial and expanded access. Lancet 399(10322):372\u2013383","journal-title":"Lancet"},{"issue":"7","key":"12322_CR50","doi-asserted-by":"crossref","first-page":"1146","DOI":"10.1002\/acn3.51796","volume":"10","author":"M Al-Saady","year":"2023","unstructured":"Al-Saady M et al (2023) Neurodegenerative disease after hematopoietic stem cell transplantation in metachromatic leukodystrophy. Ann Clin Transl Neurol 10(7):1146\u20131159","journal-title":"Ann Clin Transl Neurol"}],"container-title":["Journal of Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00415-024-12322-3.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s00415-024-12322-3\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00415-024-12322-3.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,7,9]],"date-time":"2024-07-09T10:24:05Z","timestamp":1720520645000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s00415-024-12322-3"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,4,2]]},"references-count":50,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2024,7]]}},"alternative-id":["12322"],"URL":"https:\/\/doi.org\/10.1007\/s00415-024-12322-3","relation":{},"ISSN":["0340-5354","1432-1459"],"issn-type":[{"type":"print","value":"0340-5354"},{"type":"electronic","value":"1432-1459"}],"subject":[],"published":{"date-parts":[[2024,4,2]]},"assertion":[{"value":"1 December 2023","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"5 March 2024","order":2,"name":"revised","label":"Revised","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"10 March 2024","order":3,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"2 April 2024","order":4,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"SB, MAW, SN, and AFJEV report no competing interests. JJB consulting Bluerock, Sanofi, Sobi, Omeros, SmartImmune, Immusoft, Advanced Clinical. CL is advisor in Orchard and Bluebird Bio expertise panels. MB is co-investigator to Cerevance and Covance, both within projects or remyelinating compounds and without personal payment. MSvdK is co-investigator to Ionis (trial in Alexander disease); and consultant to Calico, all without personal payment. She has a patent P112686CA00, therapeutic effects of Guanabenz treatment in vanishing white matter, to VU University Medical Center. NIW is advisor and\/or co-investigator for trials in Metachromatic Leukodystrophy and other leukodystrophies (Shire\/Takeda, Orchard, Ionis, PassageBio, VigilNeuro, Sana Biotech), without personal payment.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}},{"value":"This study was approved by the Institutional Review Board of Amsterdam UMC, and patients\/guardians gave written informed consent.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical approval"}}]}}