Chronic neuropathic pain management in spinal cord injury patients. What is the efficacy of pharmacological treatments with a general mode of administration? (oral, transdermal, intravenous).

International audienceINTRODUCTION: The pharmacological treatment of patients with spinal cord injury (SCI) pain remains challenging despite new available drugs. Such treatment should always be viewed in the context of global pain management in these patients. To date few clinical trials have been specifically devoted to this topic, and the implementation of treatments is generally based on results obtained in peripheral neuropathic pain. The aim of this review is to present evidence for efficacy and tolerability of pharmacological treatments in SCI pain and propose therapeutic recommendations. MATERIAL AND METHODS: The methodology follows the guidelines of the French Society of Physical Medicine and Rehabilitation (SOFMER). It includes a systematic review of the litterature which is performed by two independent experts. The selected studies are analysed and classified into four levels of evidence (1 to 4) and three grades of recommendations are proposed (A, B, C). The review is further validated by a reading committee. RESULTS: The efficacy of pregabalin has been confirmed in neuropathic pain associated with SCI (grade A). Gabapentin has a lower level of evidence in SCI pain (grade B) but a grade A level of evidence for efficacy in peripheral neuropathic pain. Both drugs can be proposed as first line therapy and are safe to use. Tricyclic antidepressants (TCAs) can also be proposed first line (grade B for SCI pain associated with depression, grade A for other neuropathic pain conditions), especially in patients with comorbid depressive symptoms. Tramadol can be proposed alone or in combination with antiepileptic drugs if the pain has a predominant non-neuropathic component. If these treatments fail, strong opioids can be proposed as second/third line (grade B in SCI, grade A in other types of neuropathic pain). Lamotrigine may also be proposed at this stage, particularly in patients with incomplete SCI associated with allodynia (grade B). In refractory central pain, cannabinoids may be proposed on the basis of positive results in other central pain conditions (e.g. multiple sclerosis). Intravenous ketamine and lidocaine can only be proposed in specialized centers. Drug combinations may be envisaged in case of partial response to first or second line therapy. CONCLUSIONS: Very few pharmacological studies have dealt specifically with neuropathic pain related to SCI. Large scale studies and trials comparing several active drugs are warranted in SCI pain

Convergence of patient- and physician-reported outcomes in the French National Registry of Facioscapulohumeral Dystrophy

International audienceFacioscapulohumeral muscular dystrophy (FSHD) is among the most prevalent muscular dystrophies and currently has no treatment. Clinical and genetic heterogeneity are the main challenges to a full comprehension of the physiopathological mechanism. Improving our knowledge of FSHD is crucial to the development of future therapeutic trials and standards of care. National FSHD registries have been set up to this end. The French National Registry of FSHD combines a clinical evaluation form (CEF) and a self-report questionnaire (SRQ), filled out by a physician with expertise in neuromuscular dystrophies and by the patient, respectively. Aside from favoring recruitment, our strategy was devised to improve data quality. Indeed, the pairwise comparison of data from 281 patients for 39 items allowed for evaluating data accuracy. Kappa or intra-class coefficient (ICC) values were calculated to determine the correlation between answers provided in both the CEF and SRQ. Results Patients and physicians agreed on a majority of questions common to the SRQ and CEF (24 out of 39). Demographic, diagnosis- and care-related questions were generally answered consistently by the patient and the medical practitioner (kappa or ICC values of most items in these groups were greater than 0.8). Muscle function-related items, i.e. FSHD-specific signs, showed an overall medium to poor correlation between data provided in the two forms; the distribution of agreements in this section was markedly spread out and ranged from poor to good. In particular, there was very little agreement regarding the assessment of facial motricity and the presence of a winged scapula. However, patients and physicians agreed very well on the Vignos and Brooke scores. The report of symptoms not specific to FSHD showed general poor consistency. Conclusions Patient and physician answers are largely concordant when addressing quantitative and objective items. Consequently, we updated collection forms by relying more on patient-reported data where appropriate. We hope the revised forms will reduce data collection time while ensuring the same quality standard. With the advent of artificial intelligence and automated decision-making, high-quality and reliable data are critical to develop top-performing algorithms to improve diagnosis, care, and evaluate the efficiency of upcoming treatments