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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.
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Doença Enxerto-Hospedeiro , Transtornos de Enxaqueca , Adulto , Anticorpos Monoclonais Humanizados , Antagonistas do Receptor do Peptídeo Relacionado ao Gene de Calcitonina/uso terapêutico , Método Duplo-Cego , Feminino , Doença Enxerto-Hospedeiro/tratamento farmacológico , Humanos , Masculino , Transtornos de Enxaqueca/tratamento farmacológico , Transtornos de Enxaqueca/epidemiologia , Transtornos de Enxaqueca/prevenção & controle , Estudos Retrospectivos , Resultado do Tratamento , Emirados Árabes Unidos/epidemiologiaRESUMO
INTRODUCTION: Migraine is a recurrent, disabling neurological disorder with a substantial global disease burden. However, limited real-world data are available on the patient characteristics, treatment patterns, comorbidities, and economic burden of migraine in the United Arab Emirates (UAE). In this study, we evaluated the disease burden, comorbidities, treatment patterns, specialties involved in migraine diagnosis, and healthcare resource utilization (HCRU) and associated costs in patients with migraine in Dubai, UAE. METHODS: A retrospective, secondary database cohort study was conducted from 01 January 2014 to 31 March 2022 using the Dubai Real-World Database. Patients aged ≥ 18 years with at least one diagnosis claim for migraine with continuous enrollment during the study period were included. Patients were stratified into treatment sub-cohorts. Outcomes were evaluated in terms of clinical characteristics, comorbidities, specialists visited, treatment patterns, and HCRU. RESULTS: The study included 203,222 patients (mean age: 40 years), with male predominance (55.4%). About 13.4% of patients had specific cardiovascular comorbidities. Frequently prescribed drug classes were nonsteroidal anti-inflammatory drugs (84.4%), triptans (29.8%), and beta-blockers (12.8%), while only 1.0% of patients with migraine were prescribed newer medications like calcitonin gene-related peptide antagonists. General medicine was the most frequently visited specialty on the index date (51.5%). The all-cause and migraine-specific median gross costs during the 12-month post-index period were US $1252.6 (2.4-564,740.7) and US $198.1 (0-168,903.3) respectively, with maximum contribution from inpatients. The contribution of migraine-specific median costs to all-cause median costs was highest for the diagnosis-related group (64.9%), followed by consumables (35.2%), medications (32.0%), procedures (24.5%), and services (24.5%). CONCLUSION: Migraine significantly impacts healthcare costs in the UAE. The role of newer therapies in migraine management should be explored to reduce the associated socioeconomic burden and improve patients' quality of life.
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Anti-Ma2 encephalitis is an autoimmune disorder that typically involves the brainstem, limbic system, and diencephalon. It can be paraneoplastic and is more common in males. We describe an unusual presentation of anti-Ma2 encephalitis in a patient with an XY chromosome and a female phenotype. She experienced various neurological symptoms, including olfactory hallucinations, episodic nausea, per-ictal water drinking, and hypersomnolence, that were poorly controlled by antiseizure medications (ASMs) and immunotherapy. Brain MRI showed abnormalities in right medial temporal and frontal regions, and blood tests detected anti-Ma2 antibodies. Screening for malignancies yielded no tumors. Pelvic CT showed bilateral inguinal masses and the absence of a uterus, while genetic studies revealed an XY karyotype. Surgical removal of the masses, shown to be primitive gonads, offered temporary relief, necessitating ongoing ASMs and immunotherapy.
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INTRODUCTION: The clinical, social, and economic burden of epilepsy is undeniable. Local guidance on epilepsy management is limited and needed to address the both use of anti-seizure medication (ASM) and switching practices which influence clinical outcomes. AREAS COVERED: An expert panel composed of practicing neurologists and epileptologists from countries of the Gulf Cooperation Council (GCC) met in 2022 to discuss local challenges in the management of epilepsy and formulate recommendations for clinical practice. Published literature on the outcomes of ASM switching was reviewed along with clinical practice/gaps, international guidelines, and local treatment availabilities. EXPERT OPINION: Improper ASM use and inappropriate brand-name-to-generic or generic-to-generic switching can contribute to worsening clinical outcomes in epilepsy. ASMs should be used for the management of epilepsy based on patient clinical profile, underlying epilepsy syndrome, and drug availability to ensure optimal and sustainable treatment. Both first-generation and newer ASMs can be considered; appropriate use is recommended from the beginning of treatment. It is critical to avoid inappropriate ASM switching to avoid breakthrough seizures. All generic ASMs should fulfill strict regulatory requirements. If needed, ASM changes should always be approved by the treating physician. ASM switching (brand-name-to-generic, generic-to-generic, generic-to-brand-name) should be avoided in epilepsy patients who have achieved control but can be considered for those uncontrolled on current medication.
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INTRODUCTION: Inconvenient administration and side effects of some disease-modifying therapies (DMTs) for relapsing multiple sclerosis (RMS) can deter adherence. We evaluated treatment satisfaction with cladribine tablets (CladT) for RMS in the Arabian Gulf. METHODS: This was a non-interventional, multicentre, prospective observational study in non-pregnant/lactating adults (aged ≥ 18 years) with RMS eligible for 1st treatment with CladT (EU labelling). The primary outcome was overall treatment satisfaction at 6 months (Treatment Satisfaction Questionnaire for Medication [TSQM]-14, v. 1.4), Global Satisfaction subscale. Secondary endpoints were TSQM-14 scores for convenience, satisfaction with side effects and satisfaction with effectiveness. Patients provided written informed consent. RESULTS: Of 63 patients screened, 58 received CladT and 55 completed the study. Mean age was 33 ± 9 years; mean weight 73 ± 17 kg; 31% male/69% female; mostly from the United Arab Emirates (52%) or Kuwait (30%). All had RMS (mean 0.9 ± 1.1 relapses in the past year), mean Expanded Disability Status Scale (EDSS) 1.4 ± 1.2; 36% were DMT-naïve. Mean [95% CI] score was high for overall treatment satisfaction (77.8 [73.0-82.6]), ease of use (87.4 [83.7-91.0]), tolerability (94.2 [91.0-97.3]) and effectiveness (76.2 [71.6-80.7]). Scores were similar irrespective of DMT history, age, gender, relapse history or EDSS. No relapses or serious treatment-emergent adverse events (TEAE) occurred. Two severe TEAE occurred (fatigue, headache) and 16% reported lymphopenia (two cases of grade 3 lymphopenia). Absolute lymphocyte counts at baseline and 6 months were 2.2 ± 0.8 × 109/L and 1.3 ± 0.3 × 109/L, respectively. CONCLUSIONS: Treatment satisfaction, ease of use, tolerability and patient-perceived effectiveness for CladT were high, irrespective of baseline demographics, disease characteristics and prior treatment.
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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.
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Doença de Depósito de Glicogênio Tipo II , Adulto , Criança , Consenso , Doença de Depósito de Glicogênio Tipo II/diagnóstico , Processos Grupais , HumanosRESUMO
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.
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Imunossupressores/uso terapêutico , Adesão à Medicação , Esclerose Múltipla Recidivante-Remitente/tratamento farmacológico , Prova Pericial , Humanos , Emirados Árabes UnidosRESUMO
We report the case of a 59-year-old Arab woman who was presented with acute onset of neck pain followed by quadriparesis, paraesthesias of lower limbs and incontinence of urine. Examination revealed asymmetric sensorimotor quadriparesis with sensory level at T1, establishing a clinical diagnosis of transverse myelitis. Cervical and thoracic spinal MRI showed enhancing T2/fluid attenuated inversion recovery (FLAIR) hyperintense lesion extending from C4 to C7 level in addition to long-segment lesion extending the whole of the spinal cord. She was known to have rheumatoid arthritis for the past 20 years and has been on etanercept for the past 8 years and methotrexate since past 3 years. Etanercept was stopped and she was treated with methylprednisolone followed by oral steroids and physiotherapy with which she had near complete recovery.
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Antirreumáticos/efeitos adversos , Etanercepte/efeitos adversos , Mielite Transversa/induzido quimicamente , Anti-Inflamatórios/administração & dosagem , Artrite Reumatoide/complicações , Artrite Reumatoide/tratamento farmacológico , Medula Cervical/diagnóstico por imagem , Feminino , Humanos , Pessoa de Meia-Idade , Mielite Transversa/tratamento farmacológico , Cervicalgia/etiologia , Prednisolona/administração & dosagem , Prednisolona/análogos & derivadosRESUMO
Onset of epilepsy can occur at any age, but it is relatively rare in the elderly. Late onset epilepsy is usually secondary to stroke, tumour, trauma or neurodegenerative disorders. A 62-year-old Indian woman presented with frequent drop attacks sometimes leading to unconsciousness and, rarely, associated with seizure. Her epilepsy work up was unremarkable. As the disease progressed, she was diagnosed as having idiopathic epilepsy, syncope or pseudo-seizure, on different occasions, and was treated at length with no response. Finally, detailed history-taking revealed her as having glossopharyngeal neuralgia leading to syncope and seizures. She subsequently improved. In clinical practice, such rare entities should also be considered for proper management of patients' ailments.
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Nervo Glossofaríngeo/patologia , Ossificação Heterotópica/complicações , Ossificação Heterotópica/diagnóstico , Convulsões/etiologia , Síncope/etiologia , Osso Temporal/anormalidades , Analgésicos não Narcóticos/uso terapêutico , Anticonvulsivantes/uso terapêutico , Carbamazepina/uso terapêutico , Diagnóstico Diferencial , Feminino , Humanos , Anamnese , Pessoa de Meia-Idade , Ossificação Heterotópica/fisiopatologia , Pregabalina/uso terapêutico , Osso Temporal/fisiopatologia , Resultado do TratamentoRESUMO
Involuntary movement disorders are not a common presentation of basal ganglia ischemia which may be induced by cerebral hemodynamic insufficiency. In secondary causes of movements disorders cerebrovascular diseases represent up to 22% and involuntary movements develop after 1-4% of strokes. We describe a case of a middle-aged woman who presented with intermittent involuntary tonic spasms or seizure-like episodes followed by weakness due to contralateral putaminal infarction. Initially thought to have Todd's paralysis she was not thrombolysed, but later she developed dense hemiplegia. Flexor spasms are generally thought to occur in lesions of the spinal cord but they can also occur in cerebral lesion, may be because of disinhibition of the spinal cord. Certain other theories also have been narrated, but this field still needs to be worked upon.