Real-world experience of erenumab in patients with chronic or episodic migraine in the UAE.

BACKGROUND: Erenumab is a fully human monoclonal antibody and a highly potent, first-in-class calcitonin gene-related peptide receptor inhibitor approved for migraine prevention in adults. Randomised, placebo-controlled trials show that erenumab treatment results in clinically meaningful responses, including significant reductions in monthly migraine days. Real-world evidence of the effectiveness of erenumab in patients with migraine is accruing, but gaps remain, and findings may vary according to region. We evaluated the usage patterns and effectiveness of erenumab in real-world settings in patients with migraine in the United Arab Emirates (UAE). METHODS: This retrospective, observational real-world study enrolled patients ≥ 18 years with migraine who were prescribed erenumab in the UAE. Data were collected at baseline and Months 1, 3 and 6. The primary study objective was to characterise usage patterns of erenumab in patients with chronic migraine (CM) or episodic migraine (EM) in real-world settings in the UAE. RESULTS: Of the 166 patients, 124 (74.7%) were females. The mean (standard deviation) age at migraine onset was 29 (7.93) years. Seventy-one patients (42.8%) had CM and 95 (57.2%) had EM. In the overall population, the mean monthly headache/migraine days (MHD) at baseline was 15.7 (8.45) and mean change from baseline was - 8.2 (8.83) at Month 1, - 11.0 (9.15) at Month 3 and - 11.3 (8.90) at Month 6. The mean change from baseline in monthly acute migraine-specific medication days (MSMD) was - 9.0 (8.07) at Month 1, - 9.7 (8.73) at Month 3 and - 10.7 (8.95) at Month 6. At all time points, most patients achieved at least 50% reduction in MHD (80%-91%) and MSMD (84%-94%). Similar reductions in MHD and MSMD and clinical benefit in CM or EM were seen with erenumab monotherapy or erenumab add-on therapy, with or without dose escalation and for treatment naïve or ≥ 1 previous preventive treatment failures, with additional clinical benefit in the erenumab add-on therapy and dose escalation to 140 mg subgroups. CONCLUSION: In this real-world study on erenumab use in the UAE, patients prescribed erenumab achieved clinically meaningful reductions in MHD and MSMD at all assessed time points. Erenumab was well tolerated with no new safety events.

Disease modifying treatment guidelines for multiple sclerosis in the United Arab Emirates.

The newly constituted National Multiple Sclerosis (MS) Society (NMSS)of the United Arab Emirates (UAE), set up a scientific committee to create a MS disease modifying treatment (DMT) guideline for UAE. The committee considered several unique features of the MS community in UAE including large number of expatriate population, wide variations in health insurance coverage, physician and patient preferences for DMT. The overall goal of the treatment guideline is to facilitate the most appropriate DMT to the widest number of patients. To this end it has adapted recommendations from various health systems and regulatory authorities into a pragmatic amalgamation of best practices from across the world. Importantly where data is unavailable or controversial, a common sense approach is taken rather than leave physicians and patients in limbo. The committee classifies MS into subcategories and suggests appropriate treatment choices. It recommends treatment of RIS and CIS with poor prognostic factors. It largely equates the efficacy and safety of DMT with similar mechanisms of action or drug classes e.g. ocrelizumab is similar to rituximab. It allows early switching of treatment for unambiguous disease activity and those with progression independent of relapses. Autologous hematopoietic stem cell transplantation can be offered to patients who fail one high efficacy DMT. Pragmatic guidance on switching and stopping DMT, DMT choices in pregnancy, lactation and pediatric MS have been included. It is expected that these guidelines will be updated periodically as new data becomes available.

A Multidisciplinary Perspective Addressing the Diagnostic Challenges of Late-Onset Pompe Disease in the Arabian Peninsula Region Developed From an Expert Group Meeting.

Pompe disease is a rare, metabolic, autosomal recessive disorder. Early diagnosis is critical for progressive Pompe disease as delays can significantly alter the clinical course of the disease. Diagnostic modalities, including dried blood spot testing and genetic testing, are available and are effective for diagnosing patients with late-onset Pompe disease (LOPD). However, clinicians face numerous clinical challenges related to the diagnosis of the disease. Two expert group committee meetings, involving 11 experts from the United Arab Emirates, Kuwait, the Kingdom of Saudi Arabia, and Oman, were convened in October 2019 and November 2020 respectively to develop a uniform diagnostic algorithm for the diagnosis of pediatric and adult LOPD in the Arabian Peninsula region. During the first meeting, the specialty-specific clinical presentation of LOPD was defined. During the second meeting, a diagnostic algorithm was developed after a thorough validation of clinical presentation or symptoms, which was performed with the aid of existing literature and expert judgement. A consensus was reached on the diagnostic algorithm for field specialists, such as neurologists, rheumatologists, general practitioners/internal medicine specialists, orthopedic specialists, and pulmonologists. This specialty-specific diagnostic referral algorithm for pediatric and adult LOPD will guide clinicians in the differential diagnosis of LOPD.

High-efficacy therapies for relapsing-remitting multiple sclerosis: implications for adherence. An expert opinion from the United Arab Emirates.

The number of disease-modifying treatments (DMDs) for relapsing-remitting multiple sclerosis has increased. DMDs differ not only in their efficacy and safety/tolerability, but also in the treatment burden of, associated with their initiation, route/frequency of administration, maintenance treatment and monitoring. High-efficacy DMDs bring the prospect of improved suppression of relapses and progression of disability, but may have serious safety issues, and burdensome long-term monitoring. Studies of patient preferences in this area have focused on side effects, efficacy and route of administration. Adherence to DMDs is often suboptimal in relapsing-remitting multiple sclerosis and there is a need to understand more about how the complex therapeutic and administration profiles of newer DMDs interact with these barriers to support optimal adherence to therapy.

Challenging neurological presentations of varicella virus infections in Sudan: Clinical features, imaging and recommendations.

Consequences of varicella zoster infection reflect a wide range of minor to serious complications involving the central nervous system. This is a case series with challenging presentations, imaging features and favorable outcome. For example, the cases presented with clinical features that resemble Brown Sequard Syndrome, transverse myelitis manner and multiple sclerosis and cognitive decline. We recommend adequate history taking, clinical examination and use of available investigation. Early treatment is likely to prevent any disabling neurological damage.

Fc gamma RIIa (CD32) polymorphism and onchocercal skin disease: implications for the development of severe reactive onchodermatitis (ROD).

The pathologic manifestations of Onchocerca volvulus infection depend on the interplay between the host and the parasite. A genetic single nucleotide polymorphism in the Fc gamma RIIa gene, resulting in arginine (R) or histidine (H) at position 131, affects the binding to the different IgG subclasses and may influence the clinical variations seen in onchocerciasis. This study investigated the relationship between this polymorphism and disease outcome. Fc gamma RIIa genotyping was performed on clinically characterized onchocerciasis patients (N = 100) and healthy controls (N = 74). Fc gamma RIIa genotype R/R131 frequencies were significantly higher among patients with severe dermatopathology (P < 0.001). Increased risk of developing this form was mostly associated with one tribe (Masalit) (OR = 3.2, 95% CI 1-9.9, P = 0.042). The H131 allele was found to be significantly associated with a reduced risk of having the severe form of the disease (adjusted OR = 0.26, 95% CI = 0.13-0.46, P < 0.001). Our findings suggest that the polymorphism influences the clinical outcome of onchocerciasis.