Effectiveness and safety of early intramuscular botulinum toxin injections to prevent shoulder deformity in babies with brachial plexus birth injury (POPB-TOX), a randomised controlled trial: study protocol.

INTRODUCTION: In children with brachial plexus birth injury (BPBI), denervation of the shoulder muscles leads to bony deformity in the first months of life, reducing active and passive range of motion (ROM) and causing activity limitation. The aim of this multicentre randomised controlled trial is to evaluate the effectiveness of botulinum toxin injections (BTI) in the shoulder internal rotator muscles of 12-month-old babies in limiting the progression of posterior subluxation of the glenohumeral joint, compared with a sham procedure mimicking BTI. The secondary aims are to evaluate the effectiveness of BTI in (1) limiting the progression of glenoid retroversion and three-dimensional (3D) deformity and (2) improving shoulder ROM and upper limb function, as well as to confirm the tolerance of BTI. METHODS AND ANALYSIS: Sixty-two babies with unilateral BPBI and a risk of posterior humeral head subluxation will be included. Only those with at least 7% posterior subluxation of the humeral head compared with the contralateral shoulder on the MRI will be randomised to one of two groups: 'BTI' and 'Sham'. The BTI group will receive BOTOX injections at the age of 12 months in the internal shoulder rotator muscles (8 UI/kg). The sham group will undergo a sham BTI procedure. Both groups will undergo repeated shoulder MRI at 18 months of age to quantify changes in the percentage of posterior migration of the humeral head (primary outcome), glenoid version and 3D bone deformity. Clinical evaluations (passive shoulder ROM, active movement scale) will be carried out at baseline and 15 and 18 months of age. The mini-assisting hand assessment will be rated between 10 and 11 months and at 18 months of age. Adverse events will be recorded at least monthly for each child. ETHICS AND DISSEMINATION: Full ethical approval for this study has been obtained. The findings will be disseminated in peer-reviewed publications. TRIAL REGISTRATION NUMBER: EudraCT: 2015-001402-34 in European Clinical Trial database; NCT03198702 in Clinical Trial database; Pre-results.

Unité Rhumatologique des Affections de la Main (URAM) scale: development and validation of a tool to assess Dupuytren's disease-specific disability.

OBJECTIVE: To our knowledge, no functional outcome measure has been developed and validated for Dupuytren's disease. We aimed to develop and validate a patient-reported functional outcome measure for Dupuytren's disease. METHODS: Patients with Dupuytren's disease (n = 9) and medical experts (n = 7) provided input and opinions about limiting activities that were difficult to perform because of Dupuytren's disease for item generation. The provisional scale was studied in an independent sample of patients (n = 85) for item reduction according to response distribution, reliability, redundancy, and loading in a 1-factor solution. The final scale was evaluated as follows: reliability using Cronbach's alpha coefficient and test-retest intraclass correlation coefficient from the previous 85-patient population, and construct validity and responsiveness after needle aponeurotomy in another independent 53-patient sample. For construct validity, convergent validity and divergent validity were tested. The clinically important change was estimated relative to a 1-point categorical change on the Tubiana scale. RESULTS: A 52-item provisional scale was generated and reduced to the final 9-item scale called the Unité Rhumatologique des Affections de la Main (URAM) scale (total score 0-45). The scale showed good to excellent reliability and suitable construct validity. The URAM score improved after needle aponeurotomy: the standardized effect size was 0.56. The estimated clinically important change of the URAM scale was 2.9 points. CONCLUSION: We provide the first patient-reported functional measure for Dupuytren's disease. The URAM scale demonstrated suitable psychometric properties, and is short and convenient enough for easy use in daily practice and in clinical studies.

Relation of inflammatory modic changes to intradiscal steroid injection outcome in chronic low back pain.

The purpose of this study was to analyze the association between the severity of inflammatory endplate changes (Modic) on MRI and the clinical response to intradiscal injection of corticosteroids (IDIC) in chronic low back pain (LBP). A total of 74 patients with LBP and inflammatory Modic changes who showed no response to 3-month conservative treatment received lumbar IDIC. Two spine specialists and a radiologist assessed independently the endplate marrow changes of the injected discs. We classified patients with a pure edema endplate changes on MRI as Modic type I (n = 37); those with a mixture of Modic type I and type II changes but predominantly edema changes as Modic I-2 (n = 25) and those with predominantly fatty changes as Modic II-1 (n = 12). The primary outcome was the change in LBP intensity between baseline and 1 month after IDIC, as measured on a visual analog scale (range 0-100 mm). At 1 month, reduction in pain score was significantly higher in the Modic I and Modic I-2 groups than in the Modic II-1 group (30.2 +/- 26.6 and 29.4 +/- 21.5 vs 5.3 +/- 25.5; P = 0.009 and P = 0.017, respectively). At 3 and 6 months, IDIC tended to be more effective in the Modic I and Modic I-2 groups but not significantly. No complications such as infection or hematoma were reported. IDIC could be a short-term efficient treatment for patients with chronic LBP and predominantly inflammatory endplate changes when conservative treatments have failed.