International Guidelines for the Treatment of Huntington's Disease

Abstract

The European Huntington's Disease Network (EHDN) commissioned an international task force to provide global evidence-based recommendations for everyday clinical practice for treatment of Huntington's disease (HD). The objectives of such guidelines are to standardize pharmacological, surgical and non-pharmacological treatment regimen and improve care and quality of life of patients. A formalized consensus method, adapted from the French Health Authority recommendations was used. First, national committees (French and English Experts) reviewed all studies published between 1965 and 2015 included dealing with HD symptoms classified in motor, cognitive, psychiatric, and somatic categories. Quality grades were attributed to these studies based on levels of scientific evidence. Provisional recommendations were formulated based on the strength and the accumulation of scientific evidence available. When evidence was not available, recommendations were framed based on professional agreement. A European Steering committee supervised the writing of the final recommendations through a consensus process involving two rounds of online questionnaire completion with international multidisciplinary HD health professionals. Patients' associations were invited to review the guidelines including the HD symptoms. Two hundred and nineteen statements were retained in the final guidelines. We suggest to use this adapted method associating evidence base–medicine and expert consensus to other rare diseases.

Androgen Therapy in Neurodegenerative Diseases

“…In R6/HD mice, T administration rescued AR density and functionality in the dentate gyrus [ 249 ] and in testes improved testicular function and spermatogenesis [ 258 ]. Considering that in patients with HD, therapies are symptomatic and limited to antidopaminergic neuroleptics and antidepressant drugs to regulate motor dysfunctions and mood disorders [ 259 , 260 ], androgen therapy may represent a valuable tool not yet explored. At this time, there is no documented evidence arising from human clinical trials of T treatment of patients with HD.…”

Psychiatric morbidity and poor follow-up underlie suboptimal functional and survival outcomes in Huntington’s disease

“…Advances in genetic diagnostics will add to the number of 'identifiable' conditions in the near future, but specific treatments informed by this research are still a long way away. In the meantime, symptomatic treatments in line with international guidelines recommended by Bachoud-Lévi et al, [33], specialised counselling for different category of CAG repeats, especially in the wake of new advances in gene modifiers as recommended by Migliore et al, [34], adequate rehabilitative services that improve the quality of life, and overall outcomes, need to be developed and sustained.…”

Psychological Interventions for People with Huntington’s Disease: A Call to Arms

“…The current conceptualisation and assessment of apathy in HD relies heavily on a neuropsychiatric framework, which sees it solely as the result of neurodegeneration [75]. However, as patients' understanding of their difficulties has been shown to represent a significant determinant of apathy in people with Parkinson's disease [76], similar adaptive reasons have also been recently suggested for HD [77]. Nonetheless, no investigations have been carried out so far to address apathy in pwHD with a psychological intervention.…”