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Early clinical course after hematopoietic stem cell transplantation in children with juvenile metachromatic leukodystrophy

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Early clinical course after hematopoietic stem cell transplantation in children with juvenile metachromatic leukodystrophy. / Beschle, Judith; Döring, Michaela; Kehrer, Christiane; Raabe, Christa; Bayha, Ute; Strölin, Manuel; Böhringer, Judith; Bevot, Andrea; Kaiser, Nadja; Bender, Benjamin; Grimm, Alexander; Lang, Peter; Müller, Ingo; Krägeloh-Mann, Ingeborg; Groeschel, Samuel.

In: Molecular and cellular pediatrics, Vol. 7, No. 1, 03.09.2020, p. 12.

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

Harvard

Beschle, J, Döring, M, Kehrer, C, Raabe, C, Bayha, U, Strölin, M, Böhringer, J, Bevot, A, Kaiser, N, Bender, B, Grimm, A, Lang, P, Müller, I, Krägeloh-Mann, I & Groeschel, S 2020, 'Early clinical course after hematopoietic stem cell transplantation in children with juvenile metachromatic leukodystrophy', Molecular and cellular pediatrics, vol. 7, no. 1, pp. 12. https://doi.org/10.1186/s40348-020-00103-7

APA

Beschle, J., Döring, M., Kehrer, C., Raabe, C., Bayha, U., Strölin, M., Böhringer, J., Bevot, A., Kaiser, N., Bender, B., Grimm, A., Lang, P., Müller, I., Krägeloh-Mann, I., & Groeschel, S. (2020). Early clinical course after hematopoietic stem cell transplantation in children with juvenile metachromatic leukodystrophy. Molecular and cellular pediatrics, 7(1), 12. https://doi.org/10.1186/s40348-020-00103-7

Vancouver

Beschle J, Döring M, Kehrer C, Raabe C, Bayha U, Strölin M et al. Early clinical course after hematopoietic stem cell transplantation in children with juvenile metachromatic leukodystrophy. Molecular and cellular pediatrics. 2020 Sep 3;7(1):12. https://doi.org/10.1186/s40348-020-00103-7

Bibtex

@article{7d917f63e249401aafa163b0ae1d9590,
title = "Early clinical course after hematopoietic stem cell transplantation in children with juvenile metachromatic leukodystrophy",
abstract = "BACKGROUND: Long-term outcomes of hematopoietic stem cell transplantation (HSCT) in children with juvenile metachromatic leukodystrophy (MLD) have been investigated systematically, while short-term effects of HSCT on the course of the disease remain to be elucidated.RESULTS: In this study, the clinical course was evaluated over the first 24 months following HSCT, conducted at our center in 12 children with juvenile MLD (mean follow-up 6.75 years, range 3-13.5) and compared with 35 non-transplanted children with juvenile MLD. Motor function (GMFM-88 and GMFC-MLD), cognitive function (FSIQ), peripheral neuropathy (tibial nerve conduction velocity), and cerebral changes (MLD-MR severity score) were tested prospectively. Seven children remained neurologically stable over a long period, five exhibited rapid disease progression over the first 12 to 18 months after transplantation. In the latter, time from first gross motor symptoms to loss of independent walking was significantly shorter compared with non-transplanted patients at the same stage of disease (p < 0.02). Positive prognostic factors were good motor function (GMFM = 100%, GMFC-MLD = 0) and a low MR severity score (≤ 17) at the time of HSCT.CONCLUSIONS: Our results show that if disease progression occurs, this happens early on after HSCT and proceeds faster than in non-transplanted children with juvenile MLD, indicating that HSCT may trigger disease progression.",
author = "Judith Beschle and Michaela D{\"o}ring and Christiane Kehrer and Christa Raabe and Ute Bayha and Manuel Str{\"o}lin and Judith B{\"o}hringer and Andrea Bevot and Nadja Kaiser and Benjamin Bender and Alexander Grimm and Peter Lang and Ingo M{\"u}ller and Ingeborg Kr{\"a}geloh-Mann and Samuel Groeschel",
year = "2020",
month = sep,
day = "3",
doi = "10.1186/s40348-020-00103-7",
language = "English",
volume = "7",
pages = "12",
journal = "Molecular and cellular pediatrics",
issn = "2194-7791",
publisher = "Springer Science and Business Media Deutschland GmbH",
number = "1",

}

RIS

TY - JOUR

T1 - Early clinical course after hematopoietic stem cell transplantation in children with juvenile metachromatic leukodystrophy

AU - Beschle, Judith

AU - Döring, Michaela

AU - Kehrer, Christiane

AU - Raabe, Christa

AU - Bayha, Ute

AU - Strölin, Manuel

AU - Böhringer, Judith

AU - Bevot, Andrea

AU - Kaiser, Nadja

AU - Bender, Benjamin

AU - Grimm, Alexander

AU - Lang, Peter

AU - Müller, Ingo

AU - Krägeloh-Mann, Ingeborg

AU - Groeschel, Samuel

PY - 2020/9/3

Y1 - 2020/9/3

N2 - BACKGROUND: Long-term outcomes of hematopoietic stem cell transplantation (HSCT) in children with juvenile metachromatic leukodystrophy (MLD) have been investigated systematically, while short-term effects of HSCT on the course of the disease remain to be elucidated.RESULTS: In this study, the clinical course was evaluated over the first 24 months following HSCT, conducted at our center in 12 children with juvenile MLD (mean follow-up 6.75 years, range 3-13.5) and compared with 35 non-transplanted children with juvenile MLD. Motor function (GMFM-88 and GMFC-MLD), cognitive function (FSIQ), peripheral neuropathy (tibial nerve conduction velocity), and cerebral changes (MLD-MR severity score) were tested prospectively. Seven children remained neurologically stable over a long period, five exhibited rapid disease progression over the first 12 to 18 months after transplantation. In the latter, time from first gross motor symptoms to loss of independent walking was significantly shorter compared with non-transplanted patients at the same stage of disease (p < 0.02). Positive prognostic factors were good motor function (GMFM = 100%, GMFC-MLD = 0) and a low MR severity score (≤ 17) at the time of HSCT.CONCLUSIONS: Our results show that if disease progression occurs, this happens early on after HSCT and proceeds faster than in non-transplanted children with juvenile MLD, indicating that HSCT may trigger disease progression.

AB - BACKGROUND: Long-term outcomes of hematopoietic stem cell transplantation (HSCT) in children with juvenile metachromatic leukodystrophy (MLD) have been investigated systematically, while short-term effects of HSCT on the course of the disease remain to be elucidated.RESULTS: In this study, the clinical course was evaluated over the first 24 months following HSCT, conducted at our center in 12 children with juvenile MLD (mean follow-up 6.75 years, range 3-13.5) and compared with 35 non-transplanted children with juvenile MLD. Motor function (GMFM-88 and GMFC-MLD), cognitive function (FSIQ), peripheral neuropathy (tibial nerve conduction velocity), and cerebral changes (MLD-MR severity score) were tested prospectively. Seven children remained neurologically stable over a long period, five exhibited rapid disease progression over the first 12 to 18 months after transplantation. In the latter, time from first gross motor symptoms to loss of independent walking was significantly shorter compared with non-transplanted patients at the same stage of disease (p < 0.02). Positive prognostic factors were good motor function (GMFM = 100%, GMFC-MLD = 0) and a low MR severity score (≤ 17) at the time of HSCT.CONCLUSIONS: Our results show that if disease progression occurs, this happens early on after HSCT and proceeds faster than in non-transplanted children with juvenile MLD, indicating that HSCT may trigger disease progression.

U2 - 10.1186/s40348-020-00103-7

DO - 10.1186/s40348-020-00103-7

M3 - SCORING: Journal article

C2 - 32910272

VL - 7

SP - 12

JO - Molecular and cellular pediatrics

JF - Molecular and cellular pediatrics

SN - 2194-7791

IS - 1

ER -