Evaluation of Accumulated Mucopolysaccharides in the Brain of Patients with Mucopolysaccharidoses by 1H-Magnetic Resonance Spectroscopy before and after Bone Marrow Transplantation

Takahashi, Yukitoshi; Sukegawa, Kazuko; Aoki, Masato; Ito, Akiko; Suzuki, Kiyotaka; Sakaguchi, Heima; Watanabe, Mahito; Isogai, Koji; Mizuno, Shinji; Hoshi, Hiroaki; Kuwata, Kazuo; Tomatsu, Shunji; Kato, Shingo; Ito, Tetsuhide; Kondo, Naomi; Orii, Tadao

doi:10.1203/00006450-200103000-00008

Cited by 52 publications

(34 citation statements)

References 24 publications

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“…It has recently become an additional tool in the evaluation of neurologic involvement in patients with MPS, and numerous studies have proposed the use of MR spectroscopy metabolite alterations as biomarkers demonstrating disease severity. A decade ago, Takahashi et al, 13 by using MR spectroscopy, found a resonance that was higher than that of myo-inositol and correlated this finding with in vitro GAG accumulation. Additionally, they have shown an elevation in the choline/creatine ratio in white matter lesions, speculating that this finding was the result of myelin damage, increased glial proliferation, or elevated cell membrane synthesis.…”

Section: Mr Spectroscopymentioning

confidence: 95%

“…Nevertheless, the frequency, magnitude, and time of occurrence of WMA are variable in the different forms of MPS. 9 Those changes are prominent in MPS I, [10][11][12][13][14][15][16][17][18][19] II, 12-14,16,18,20-27 IIIA, 10,11 IIIB, 18,21,28 IIID, 29 and VII, 13,19 whereas in MPS IV and VI, they are found to a lesser extent and may not become apparent until adulthood. 16,19,21,30,31 WMA mainly consist of diffuse or focal areas of prolonged T1 and T2 relaxation times.…”

Section: White and Gray Matter Signal-intensity Abnormalitiesmentioning

confidence: 99%

“…23,34 Although prominent in most patients with MPS, this feature is nonspecific because it was found in numerous pathologic conditions and healthy subjects as well. 35 Enlarged PVS have been described so far in MPS I, [10][11][12][13]15,[17][18][19]36 II, 10,12,13,18,[20][21][22][23]27,37 IIIA, 10,38,39 IIIB, 28,40,41 and VI. 16,18,19,30 In general, children with MPS I and II show the most profound enlargement of PVS, giving rise to a ''sieve-like" appearance.…”

Section: Brain Lesions and Enlarged Pvsmentioning

confidence: 99%

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Brain and Spinal MR Imaging Findings in Mucopolysaccharidoses: A Review

Zafeiriou

Batzios

2012

AJNR Am J Neuroradiol

117

106

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SUMMARY: MPS represents a group of rare hereditary disorders characterized by multisystem involvement due to intralysosomal GAG accumulation. Among various tissues, both the central and peripheral nervous system are affected in almost all types of the disease. Thus, brain and spinal MR imaging are valuable tools for the assessment of neurologic involvement, and there is evidence that they might be reliable markers demonstrating disease severity and efficacy of treatment options currently used in patients with MPS. We aimed to review the most prominent MR imaging features of patients with MPS, paying attention to the physiopathologic mechanisms responsible for these alterations. Along with the description of neuroimaging findings, existing data in relation to their correlation with the severity of neurologic involvement is discussed, while another topic of great importance is the effect of various therapeutic regimens in the progression of brain and spinal MR imaging alterations. Finally, recent data concerning MR spectroscopy studies in MPS are also critically discussed. ABBREVIATIONS:ERT ϭ enzyme replacement therapy; GAG ϭ glycosaminoglycan; HSCT ϭ hematopoietic stem cell transplantation; MPS ϭ mucopolysacchari-

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Section: Mr Spectroscopymentioning

confidence: 95%

Section: White and Gray Matter Signal-intensity Abnormalitiesmentioning

confidence: 99%

Section: Brain Lesions and Enlarged Pvsmentioning

confidence: 99%

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Brain and Spinal MR Imaging Findings in Mucopolysaccharidoses: A Review

Zafeiriou

Batzios

2012

AJNR Am J Neuroradiol

117

106

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“…[1][2][3][4][5][6][7][8] The growing number of patients who have survived childhood malignancy has increased the importance of monitoring the long-term side effects. Acute renal impairment is a major side effect of BMT, often leading to permanent renal failure.…”

Section: Introductionmentioning

confidence: 99%

Long-term renal function following bone marrow transplantation

Grönroos

Bolme

Winiarski

et al. 2007

Bone Marrow Transplant

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Renal function, evaluated as glomerular filtration rate (GFR) and effective renal plasma flow (ERPF), was investigated in 187 pediatric patients who underwent allogeneic (n ¼ 169) or autologous bone marrow transplantation (BMT). Allogeneic BMT patients were divided into three groups: hematological malignancies, aplastic anemia and non-malignant diseases, whereas autologous patients constituted a fourth group. A total of 64% received total body irradiation (TBI) as conditioning therapy, and 50 healthy children served as controls. GFR and ERPF were normal before transplantation. After 1 year, both GFR and ERPF were significantly reduced. GFR had recovered slightly after 3 years and remained stable thereafter. Recovery in ERPF was not apparent. Renal impairment was found in 41% of patients at 1 year, in 31% at 3 years and in 11% 7 years after BMT. Patients with hematological malignancies had lower GFRs than patients with non-malignant diseases at all time points. The most important risk factor as regards chronic renal impairment was TBI. Type of donor, cyclophosphamide (CY), or acute graft-versus-host disease (GVHD) did not seem to contribute to the development of chronic renal impairment. We suggest that tests of renal function should be included in long-term followup after BMT.

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“…Similarly, Takahashi et al demonstrated the effect of BMT in patients with mucopolysaccharidoses using MRS (Takahashi et al 2001). …”

mentioning

confidence: 96%

Cerebral Magnetic Resonance Spectroscopy Demonstrates Long-Term Effect of Bone Marrow Transplantation in α-Mannosidosis

2013

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a-Mannosidosis, OMIM #248500, is an autosomal recessive lysosomal storage disease caused by acidic a-mannosidase deficiency. Treatment options include bone marrow transplantation (BMT) and, possibly in the future, enzyme replacement therapy. Brain magnetic resonance spectroscopy (MRS) enables non-invasive monitoring of cerebral treatment effect. Accumulated cerebral mannosecontaining oligosaccharides were demonstrated by MRS in a patient who at age 2 years and 11 months received a BMT from a haploidentical non-carrier sibling. The cerebral mannose-containing oligosaccharides had disappeared as early as 9½ months after BMT. MRS furthermore demonstrated the persistent treatment effect at regular intervals up to 5½ years after BMT. MRS is a non-invasive tool that can demonstrate the effect of BMT treatment. Likewise, MRS may be used to demonstrate the cerebral effect of other potential treatments such as enzyme replacement therapy.

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Evaluation of Accumulated Mucopolysaccharides in the Brain of Patients with Mucopolysaccharidoses by 1H-Magnetic Resonance Spectroscopy before and after Bone Marrow Transplantation

Cited by 52 publications

References 24 publications

Brain and Spinal MR Imaging Findings in Mucopolysaccharidoses: A Review

Brain and Spinal MR Imaging Findings in Mucopolysaccharidoses: A Review

Long-term renal function following bone marrow transplantation

Cerebral Magnetic Resonance Spectroscopy Demonstrates Long-Term Effect of Bone Marrow Transplantation in α-Mannosidosis

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