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, Jahrgang 7, Nr. 1, 03.09.2020, S. 12.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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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 -