DEVELOPMENTAL MEDICINE & CHILD NEUROLOGY

ORIGINAL ARTICLE

Botulinum toxin A injections and occupational therapy in children with unilateral spastic cerebral palsy: a randomized controlled trial GIT LIDMAN 1,2

| ANN NACHEMSON 3 | MARIE PENY-DAHLSTRAND 1,2 | KATE HIMMELMANN 2,4

1 Institute of Neuroscience and Physiology at the Sahlgrenska Academy, University of Gothenburg, Gothenburg; 2 Regional Rehabilitation Center, Queen Silvia’s Children’s Hospital, Sahlgrenska University Hospital, Gothenburg; 3 Department of Hand and Plastic Surgery/Hand Surgery, Sahlgrenska University Hospital, Gothenburg; 4 Department of Paediatrics, Institute of Clinical Sciences at the Sahlgrenska Academy, University of Gothenburg, Gothenburg, Sweden. Correspondence to Git Lidman at Institute of Neuroscience and Physiology, Sahlgrenska Academy at the University of Gothenburg, Box 455, SE-405 30 G€oteborg, Sweden. E-mail: [email protected] This article is commented on by Russo on pages 702–703 of this issue.

PUBLICATION DATA

Accepted for publication 24th January 2015. Published online 22nd March 2015. ABBREVIATIONS

AHA BoNT-A/ OT COPM ICF-CY

ROM SDD USCP

Assisting Hand Assessment Botulinum toxin A plus occupational therapy Canadian Occupational Performance Measure International Classification of Functioning, Disability and Health for Children and Youth Range of movement Smallest detectable difference Unilateral spastic cerebral palsy

AIM To investigate the effects of repeated botulinum toxin A (BoNT-A) injections combined with occupational therapy, including a splint, compared with occupational therapy alone on hand function in children with unilateral spastic cerebral palsy (USCP), in all International Classification of Functioning, Disability and Health (ICF) domains. METHOD This was a randomized controlled study, population-based and evaluator-blinded for primary outcome (October 2004 to September 2010). Twenty children (14 males; median age 3y 1mo) with USCP, recruited at a rehabilitation centre in Sweden, were assigned to one of two parallel groups using concealed allocation. In the course of one year, 10 children received occupational therapy, while 10 received repeated BoNT-A plus occupational therapy (BoNT-A/OT). Primary outcome (Assisting Hand Assessment [AHA]), and secondary outcome measures (range of movement [ROM], and Canadian Occupational Performance Measure), were measured at baseline, 3, 6, 9, and 12 months. RESULTS AHA revealed a superior effect in the BoNT-A/OT group at 12 months: 6 out of 10 improved compared with 1 out of 10 in the occupational therapy group (p10°. In ROM passive extension of the elbow, at 12 months, 0 out of 10 in the BoNT-A/OT group and 1 out of 10 in the occupational therapy group had improved by >10° (Table II). Missing baseline data for active elbow extension: BoNTA/OT (n=4) and occupational therapy (n=2).

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more than 10°, according to Armstrong et al.20 A difference of two points is considered to be a clinically relevant change for the COPM according to Law et al.18 and Carswell et al.21 This concept provided an opportunity to detect a relevant change in all ICF domains. Repeated injections combined with training appeared to have a positive effect. Lowe et al. reported similar findings.8 Active supination was increased immediately after the first block of intervention, in both groups, to some extent maintained at the start of block two. Starting at a higher point in the second block of intervention, a maximum effect was seen at 9 months, mostly maintained at 12 months. No similar finding of increased active supination has been reported previously. The use of a night splint, designed to stretch the pronator muscles and used several hours per night may have contributed to the beneficial effect, especially in those who did not receive BoNT-A. A similar result was seen in the AHA in the BoNT-A/OT group in terms of increased bimanual ability. The AHA scores increased in the BoNTA/OT group and were maintained after 12 months. At 6 months, the scores were higher compared with baseline and, consequently, the participants started at a higher level in the second block. Their score still increased in the last block, indicating increasing performance over time and results lasting longer than the effects of the drug. The child with CP needs time to learn effective strategies finally to reach the autonomous stage.1 It can be speculated that the decrease in muscle tone after BoNT-A provided the window of opportunity the child needed to facilitate the use of the impaired hand and perform bimanual activities of enhanced quality, while, in the occupational therapy group, the absence of improvement was the result of sustained muscle tone. Other studies show unchanged results22 after occupational therapy, but also improvement.23 The study design often differs, making comparisons difficult.9 The majority had a maximum passive ROM at baseline, and a ceiling effect therefore occurred. Five of the six participants in the BoNT-A/OT group, who maintained their improvements in the AHA score at 12 months, also improved in active supination. This is consistent with the statement by Braendvik et al., suggesting that active supination is a contributory factor in the performance of bimanual activity.24 Six participants in the BoNT-A/OT group and three in the occupational therapy group were unable to actively supinate the forearm to 0° at baseline. Therefore, three more children had the opportunity to reach 0° in the BoNT-A/OT group. This may be one of many reasons explaining improvements in the AHA score in this group. Both groups improved in individual goal achievement measured by the COPM. Goal-setting was essential in the clinical decision-making.25 The parents prioritized the majority of their goals in the self-care category, consistent with other studies.9 The children were too small to be able to select goals themselves that were sustainable over time. Goal-directed training increased performance, regardless of the therapy model. Increased ability in a larger number 760 Developmental Medicine & Child Neurology 2015, 57: 754–761

of activities was also enhanced by improving the overall bimanual skills. In the current study, this occurred only when BoNT-A was added. Limitations of this study were the small sample, the simple randomization, and the partially unblinded design, where participants were aware of their group allocation. Only 20% of the total group with USCP in this geographical area were eligible to be part of the study, according to our detailed knowledge also of those who were not included. The small study size limits generalization of the results. The improvement in active ROM and goal performance in the occupational therapy group is difficult to interpret because of the lack of a non-treated control group; however, this was not considered to be ethical. Moreover, it is important to note that the difference in active supination between the groups at baseline may have been of clinical importance. To study the effects of early interventions on a longterm basis, the participants will be monitored for 3 years. Everyday life includes a variety of activities requiring bimanual ability. This small study compared the effectiveness of BoNT-A/OT versus occupational therapy alone for young children with unilateral CP. Both interventions demonstrated improvements in active ROM and goal performance, but BoNT-A, as an adjunct to occupational therapy, demonstrated a superior effect on bimanual performance. BoNT-A/OT may be considered when the objective is to improve bimanual ability in daily life. A CK N O W L E D G E M E N T S We sincerely thank the participating children and families, occupational therapists Kristina Olsson and Karin Lindh, and the occupational therapists at the habilitation centres. G€ oran Carlsson PhD and Catrin Wessman PhD who assisted with statistical calculations. This study was funded by the NorrbackaEugenia, the Folke Bernadotte and the Petter Silfverski€ old Memorial Foundations, the Sunnerdahl Handikappfond, and the Research and Development Boards for Gothenburg & S€ odra Bohusl€an and V€astra G€ otaland. The authors have stated they had no interests that might be perceived as posing a conflict or bias.

SUPPORTING INFORMATION The following additional material may be found online: Figure S1. Circular night splint, individually made for each child, with the elbow flexed, the forearm in supination, and the thumb between radial and volar abduction. Figure S2. (a) Results of the primary outcome for individual cases at baseline and at 12 months in the botulinum toxin A injections plus occupational therapy group. (b) Results of the primary outcome for individual cases at baseline and at 12 months in the occupational therapy group. Figure S3. (a) Active supination related to the Assisting Hand Assessment at baseline and 12 months in the botulinum toxin A injections plus occupational therapy group. (b) Active supination related to the Assisting Hand Assessment at baseline and 12 months in the occupational therapy group.

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Botulinum Toxin and Occupational Therapy in USCP Git Lidman et al.

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Botulinum toxin A injections and occupational therapy in children with unilateral spastic cerebral palsy: a randomized controlled trial.

To investigate the effects of repeated botulinum toxin A (BoNT-A) injections combined with occupational therapy, including a splint, compared with occ...
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