Old measures and new scores in spinal muscular atrophy patients

被引：5

作者：

Mazzone, Elena ^{[1
]}

Montes, Jacqueline ^{[2
]}

Main, Marion ^{[3
,4
]}

Mayhew, Anna ^{[5
]}

Ramsey, Danielle ^{[3
,4
]}

Glanzman, Allan M. ^{[6
]}

Dunaway, Sally ^{[2
]}

Salazar, Rachel ^{[2
]}

Pasternak, Amy ^{[7
]}

Quigley, Janet ^{[7
]}

Pane, Marika ^{[1
]}

Pera, Maria C. ^{[1
]}

Scoto, Mariacristina ^{[3
,4
]}

Messina, Sonia ^{[8
]}

Sframeli, Maria ^{[8
]}

D'amico, Adele ^{[9
]}

Van Den Hauwe, Marleen ^{[10
]}

Sivo, Serena ^{[1
]}

Goemans, Nathalie ^{[10
]}

Darras, Basil T. ^{[7
]}

Kaufmann, Petra ^{[2
]}

Bertini, Enrico ^{[9
]}

De Vivo, Darryl C. ^{[2
]}

Muntoni, Francesco ^{[3
,4
]}

Finkel, Richard ^{[11
]}

Mercuri, Eugenio ^{[1
,3
,4
]}

机构：

[1] Catholic Univ, Dept Paediat Neurol, I-00168 Rome, Italy

[2] Columbia Univ, Dept Neurol, Med Ctr, New York, NY USA

[3] UCL Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England

[4] Great Ormond St Hosp Sick Children, London, England

[5] Newcastle Univ, Inst Med Genet, Newcastle Upon Tyne NE1 7RU, Tyne & Wear, England

[6] Childrens Hosp Philadelphia, Dept Phys Therapy, Philadelphia, PA USA

[7] Harvard Univ, Sch Med, Dept Neurol, Boston Childrens Hosp, Boston, MA 02115 USA

[8] Univ Messina, Dept Neurosci, Messina, Italy

[9] IRCCS Bambino Gesu Childrens Hosp, Unit Neuromuscular & Neurodegenerat Disorders, Dept Neurosci, Rome, Italy

[10] Univ Hosp Leuven, Dept Child Neurol, Leuven, Belgium

[11] Univ Cent Florida, Coll Med, Nemours Childrens Hosp, Orlando, FL 32816 USA

来源：

MUSCLE & NERVE | 2015年 / 52卷 / 03期

关键词：

neuromuscular; outcome measures; Rasch analysis; scoring; spinal muscular atrophy; FUNCTIONAL MOTOR SCALE; PHENYLBUTYRATE; RELIABILITY; CHILDREN; TRIAL;

D O I：

10.1002/mus.24748

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Introduction: A recent Rasch analysis performed on the Hammersmith Functional Motor ScaleExpanded (HFMSE) in patients with spinal muscular atrophy (SMA) identified issues impacting scale validity, redundant items, and disordered thresholds on some items. Methods: We modified the HMFSE scoring based on the Rasch analysis and on expert consensus to establish whether the traditional scoring overestimated the number of patients with changes within 2 points from baseline. Data were collected retrospectively from multicenter data sets in 255 type 2 and 3 SMA patients. Results: The mean 12-month changes using the new and the traditional scoring system did not differ significantly (P>0.05). The numbers of patients who improved or decreased by >2 points were also similar. Conclusions: The presence of outliers using the traditional scoring system was not due to overestimation of changes in activities that were tested bilaterally or to discrepancies in the scoring hierarchy of individual items. Muscle Nerve 52:435-437, 2015

引用

页码：435 / 437

页数：3

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