Gross Motor Development of Children With Hurler Syndrome After Umbilical Cord Blood Transplantation

Dusing, Stacey C.; Thorpe, Deborah; Poe, Michele D.; Rosenberg, Angela; Mercer, Vicki S.; Escolar, Maria L.

doi:10.2522/ptj.20070017

Cited by 13 publications

(9 citation statements)

References 11 publications

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“…All of these studies addressed one of three aspects of gait: 1) development of independent walking in toddlers, 2) quality of gait, and 3) independent gait and mobility in late childhood/ adolescence. With respect to independent walking, four studies reported a delayed onset (Hugh-Jones 1986; Dusing et al 2007a; Ringden et al 2006; Dusing et al 2007b). This developmental delay was attributed to limited practice during hospitalisation for HSCT, in addition to orthopaedic abnormalities (Dusing et al 2007a).…”

Section: Resultsmentioning

confidence: 99%

“…Other aspects that contribute to motor functioning are the development of gross and fine motor skills. These were generally studied on a group level, using developmental scales such as the Vineland Adaptive Behavior Scales (VABS) (Bjoraker et al 2006), Peabody Developmental Motor Scales, edition 2 (PDMS-2) (Dusing et al 2007a;Dusing et al 2007b;D u s i n ge ta l .2005), Denver Developmental Screening Test (DDST) (Lucke et al 2007), and the more recently developed MPS-PPM (MPS physical performance measure) (Dumas et al 2004). Evaluation of these five studies, including a total of 63 patients, showed that Hurler patients were limited in their gross motor development and had mild to pronounced motor retardation between 0 and 4 years of age, when compared to age-referenced controls.…”

Section: Description Of Studiesmentioning

confidence: 99%

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Orthopaedic management of Hurler's disease after hematopoietic stem cell transplantation: a systematic review

Linden

Kruyt

Sakkers

et al. 2011

J of Inher Metab Disea

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ObjectiveThe introduction of hematopoietic stem cell transplantation (HSCT) has significantly improved the life-span of Hurler patients (mucopolysaccharidosis type I-H, MPS I-H). Yet, the musculoskeletal manifestations seem largely unresponsive to HSCT. In order to facilitate evidence based management, the aim of the current study was to give a systematic overview of the orthopaedic complications and motor functioning of Hurler’s patients after HSCT.MethodsA systematic review was conducted of the medical literature published from January 1981 to June 2010. Two reviewers independently assessed all eligible citations, as identified from the Pubmed and Embase databases. A pre-developed data extraction form was used to systematically collect information on the prevalence of radiological and clinical signs, and on the orthopaedic treatments and outcomes.ResultsA total of 32 studies, including 399 patient reports were identified. The most frequent musculoskeletal abnormalities were odontoid hypoplasia (72%), thoracolumbar kyphosis (81%), genu valgum (70%), hip dysplasia (90%) and carpal tunnel syndrome (63%), which were often treated surgically during the first decade of life. The overall complication rate of surgical interventions was 13.5%. Motor functioning was further hampered due to reduced joint mobility, hand dexterity, motor development and longitudinal growth.ConclusionStem cell transplantation does not halt the progression of a large range of disabling musculoskeletal abnormalities in Hurler’s disease. Although prospective data on the quantification, progression and treatment of these deformities were very limited, early surgical intervention is often advocated. Prospective data collection will be mandatory to achieve better evidence on the effect of treatment strategies.

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Section: Resultsmentioning

confidence: 99%

Section: Description Of Studiesmentioning

confidence: 99%

Orthopaedic management of Hurler's disease after hematopoietic stem cell transplantation: a systematic review

Linden

Kruyt

Sakkers

et al. 2011

J of Inher Metab Disea

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“…CD34+ cells have been used for treating malignant diseases such as MI [5–9, 18, 19], hematological malignancies [20–22], Hurler syndrome [23, 24], Krabbe disease [25], primary immunodeficiency diseases [26], bone marrow failure [27], and β‐thalassemia [28]. Along with the advantages, CD34+ cells also have limitations.…”

Section: Discussionmentioning

confidence: 99%

Mesenchymal Stem Cells and Mononuclear Cells From Cord Blood: Cotransplantation Provides a Better Effect in Treating Myocardial Infarction

Chen

Yue²,

et al. 2016

Stem Cells Translational Medicine

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The aim of this study was to evaluate the effect of cotransplanting mononuclear cells from cord blood (CB-MNCs) and mesenchymal stem cells (MSCs) as treatment for myocardial infarction (MI). Transplanting CD341 cells or MSCs separately has been shown effective in treating MI, but the effect of cotransplanting CB-MNCs and MSCs is not clear. In this study, MSCs were separated by their adherence to the tissue culture. The morphology, immunophenotype, and multilineage potential of MSCs were analyzed. CB-MNCs were separated in lymphocyte separation medium 1.077. CD341 cell count and viability were analyzed by flow cytometry. Infarcted male Sprague-Dawley rats in a specificpathogen-free grade were divided into four treatment groups randomly: group I, saline; group II, CB-MNCs; group III, MSCs; and group IV, CB-MNCs plus MSCs. The saline, and CB-MNCs and/or MSCs were injected intramyocardially in infarcted rats. Their cardiac function was evaluated by echocardiography. The myocardial capillary density was analyzed by immunohistochemistry. Both cell types induced an improvement in the left ventricular cardiac function and increased tissue cell proliferation in myocardial tissue and neoangiogenesis. However, CB-MNCs plus MSCs were more effective in reducing the infarct size and preventing ventricular remodeling. Scar tissue was reduced significantly in the CB-MNCs plus MSCs group. MSCs facilitate engraftment of CD341 cells and immunomodulation after allogeneic CD341 cell transplantation. Cotransplanting MSCs and CB-MNCs might be more effective than transplanting MSCs or CB-MNCs separately for treating MI. This study contributes knowledge toward effective treatment strategies for MI. STEM CELLS TRANSLATIONAL MEDICINE 2016;5:350-357 SIGNIFICANCEThis study assessed cotransplantation of hematopoietic stem cells (CD341 cells) and mesenchymal stem cells (MSCs) for treatment of myocardial infarction (MI) in a rat model. The results demonstrate that MSCs and mononuclear cells from cord blood may have synergistic effects and cotransplantation might be more effective in the treatment of MI than transplanting MSCs and CD341 cells separately. This study contributes knowledge toward a more effective treatment protocol for MI.

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“…Com relação à paciente estudada, ela apresentava baixo desempenho tanto na motricidade global como nas habilidades finas em relação ao esperado para a sua IC. Dados semelhantes são referidos em outros estudos sobre desenvolvimento motor na MPS tipo 1 (5,6,11,12) . A deficiência na fase de aquisição das habilidades motoras decorre das lesões do sistema central e da falta de experiências sensório-motoras, dificultando, assim, a capacidade de planejamento e, consequentemente, a execução das habilidades propostas (7,10) .…”

Section: Discussionunclassified

“…Caracteriza-se por ser doença multisistêmica e progressiva, com anormalidades ósseas, atraso no desenvolvimento neuromotor (2) , além de complicações cardíacas resultantes da elevada deposição de GAGs nas artérias coronárias e válvulas cardíacas (1,3) . Apesar do atraso motor evidente nos pacientes acometidos, os relatos acerca das características motoras e funcionais são escassos, e o conhecimento desse aspecto da doença vem aumentando mais recentemente, após o advento da intervenção medicamentosa (4)(5)(6) . A influência do tratamento fisioterapêutico na MPS tipo 1 não é descrita em pesquisas científicas, sendo tal intervenção prescrita para otimizar a funcionalidade e a autonomia de crianças portadoras da doença.…”

Section: Introductionunclassified

Evolução motora de paciente com mucopolissacaridose tipo 1

Iwabe

Frezzato

Nogueira³

2010

Rev. paul. pediatr.

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RESUMOObjetivo: Relatar os aspectos funcionais de uma criança com mucopolissacaridose tipo 1, descrevendo suas principais alterações após breve tratamento fisioterapêutico.Descrição do caso: Criança com 34 meses, portadora de mucopolissacaridose tipo 1 iniciou tratamento fisioterapêutico, duas vezes por semana, durante 50 minutos, sendo avaliada mensalmente por meio das atividades propostas pela Escala de Desenvolvimento Motor (EDM), que analisa a motricidade fina e global, o equilíbrio, o esquema corporal e a organização temporal e espacial. Na primeira avaliação, a criança apresentava-se com 34 meses de idade cronológica e 18 meses de idade motora geral, com melhor pontuação no item organização espacial, segundo a EDM. Após quatro meses, a idade motora geral era de 20 meses; porém, houve melhora da motricidade global.Comentários: A análise das atividades funcionais do paciente relatado demonstrou atraso no desenvolvimento motor. A intervenção terapêutica possibilitou evolução favorável, principalmente na motricidade global, mesmo tratando-se de um portador de doença evolutiva.Palavras-chave: deficiências do desenvolvimento; mucopolissacaridose I; desenvolvimento infantil; fisioterapia. ABSTRACT Objective:To report the functional aspects of a child with mucopolysaccharidosis type 1 and to describe its main dysfunctions after a brief physiotherapy intervention.Case description: A 34 months old child with mucopolysaccharidosis type 1 started a physiotherapy treatment twice a week (50 minutes each session). Her motor skills were monthly evaluated by the Motor Development Scale (MDS), which analyzes fine and global motor skills, balance, body schema and temporal and spatial organization. In the first evaluation, the child had 34 months of chronological age and 18 months of global motor age, with better performance in spacial organization, according to MDS. After four months, the child had 20 months of general motor age; however, the global motor skills improved.Comments: The analysis of the patient's functional activities showed motor delay. The therapeutic intervention allowed favorable outcome, mainly in the global motor skills, even considering the presence of a progressive disease.

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Gross Motor Development of Children With Hurler Syndrome After Umbilical Cord Blood Transplantation

Cited by 13 publications

References 11 publications

Orthopaedic management of Hurler's disease after hematopoietic stem cell transplantation: a systematic review

Orthopaedic management of Hurler's disease after hematopoietic stem cell transplantation: a systematic review

Mesenchymal Stem Cells and Mononuclear Cells From Cord Blood: Cotransplantation Provides a Better Effect in Treating Myocardial Infarction

Evolução motora de paciente com mucopolissacaridose tipo 1

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