Treatment Of Acute Attacks Research Articles

The use of onabotulinumtoxin A in migraine: mechanism of action, efficacy, safety and tolerability

Migraine is a common, yet disabling, neurological disorder that is characterised by recurrent attacks of headache lasting 4–72 hours at a time. Headaches may also present with associated symptoms, such as nausea, vomiting, photophobia and/or phonophobia. There is a distinction between episodic migraine (EM) and chronic migraine (CM). Classed as a distinct clinical entity, patients with CM suffer from more headache days, increased headache disability, reduced health-related quality of life and greater co-morbidity than those with EM. CM is also considered a greater socioeconomic burden and associated with higher healthcare resource utilisation compared to EM. The main goal in CM treatment is to reduce the impact of migraine on the patient's life. Treatments combine lifestyle changes, trigger management and pharmacological management based on two aims: immediate treatment of acute migraine attacks and prophylactic treatment. Onabotulinumtoxin A is the only botulinum toxin licensed for the prevention of headaches in CM patients. The efficacy of injectable onabotulinumtoxin A for headache prevention in adults with CM was evaluated over 1 year in the phase 3 clinical trials of PREEMPT 1 and PREEMPT 2. A longer term study over a period of 2 years took place, and this phase 4 study was known as COMPEL. Good clinical evidence was found that treatment with onabotulinumtoxin A leads to a reduction of monthly headache days, as well as improvement in quality of life, and was both safe and well tolerated.

Subcutaneous sumatriptans for acute migraine attacks in adults.

Migraines are common and most often described as painful pulsatile headaches with nausea and sensory sensitivities. Sumatriptan is one of many medications available for treatment of acute migraine attacks.

Optimal Dosing of Lasmiditan in the Management of Acute Migraine Attack: A Systematic Review and Meta-analysis.

Background:The current target of migraine treatment is focused on Triptans. Lasmiditan, a non-vasoconstrictive and highly selective 5HT1F receptor agonist is a novel therapeutic discovery for migraine for patients with cardiovascular (CV) risk factors or stable cardiovascular diseases and who fail to respond to the existing treatment.Objective:To identify an optimal dosing of Lasmiditan 100 mg versus 200 mg for the treatment of acute migraine attacks in adult patients with cardiovascular risk factors.Methods:Systematic searches were run in databases such as Cochrane Central Register of Controlled Trials (CENTRAL), Scopus, Google scholar, and PUBMED. Out of 83 study records identified, two studies were included for quantitative analysis.Results:There was a significant headache pain freedom at 2 h [Odds Ratio (OR): 0.77; 95% Confidence interval (CI): 0.64–0.92] and sustained pain freedom at 24 h (OR): 0.75; 95% CI: 0.61–0.93] in patients taking Lasmiditan 200 mg compared to those taking Lasmiditan 100 mg. The results were statistically insignificant for parameters like most bothersome symptoms (MBS) free at 2 h, headache relief at 2 h, disability level at 2 h, and global impression of change at 2 h. A combined analysis of these parameters showed a remarkable difference between both the groups favoring Lasmiditan 200 mg [OR: 0.88; 95% CI: 0.81–0.95].Conclusion:An oral dosing of Lasmiditan 200 mg is ideal for the treatment of acute migraine in adult patients with CV risk factors for attaining headache pain freedom at 2 h and sustained pain freedom at 24 compared to Lasmiditan 100 mg.

Cluster Headache: What's New?

Cluster headache is a highly disabling primary headache disorder which is widely described as the most painful condition a human can experience. To provide an overview of the clinical characteristics, epidemiology, risk factors, differential diagnosis, pathophysiology and treatment options of cluster headache, with a focus on recent developments in the field. Structured review of the literature on cluster headache. Cluster headache affects approximately one in 1000 of the population. It is characterised by attacks of severe unilateral head pain associated with ipsilateral cranial autonomic symptoms, and the tendency for attacks to occur with circadian and circannual periodicity. The pathophysiology of cluster headache and other primary headache disorders has recently become better understood and is thought to involve the hypothalamus and trigeminovascular system. There is good quality evidence for acute treatment of attacks with parenteral triptans and high flow oxygen; preventive treatment with verapamil; and transitional treatment with oral corticosteroids or greater occipital nerve injection. New pharmacological and neuromodulation therapies have recently been developed. Cluster headache causes distinctive symptoms, which once they are recognised can usually be managed with a variety of established treatments. Recent pathophysiological understanding has led to the development of newer pharmacological and neuromodulation therapies, which may soon become established in clinical practice.

Treatment of headache disorders with acupuncture: a 6-year retrospective study.

Existing systematic reviews and meta-analyses indicate that acupuncture has similar clinical effectiveness in the prevention of headache disorders (HDs) as drug therapy, but with fewer side effects. As such, examining acupuncture's use in a pragmatic, real-world setting would be valuable. The purpose of this study was to compare the effects of acupuncture and prophylactic drug treatment (PDT) on headache frequency in patients with HDs, under real-world clinical conditions. Retrospective cohort study of patients with HDs referred to a pain clinic, using electronic health record data. Patients continued with tertiary care (treatment of acute headache attacks and lifestyle, meditation, exercise and dietary instructions) with PDT, or received 12 sessions of acupuncture over 3 months, instead of PDT under conditions of tertiary care. The primary outcome data were the number of days with headache per month, and groups were compared at baseline and at the end of the third month of treatment. Data were analysed for 482 patients with HDs. The number of headache days per month decreased by 3.7 (standard deviation (SD) = 2.9) days in the acupuncture group versus 2.9 (SD = 2.3) in the PDT group (p = 0.007). The proportion of responders was 39.5% versus 16.3% (p < 0.001). The number needed to treat was 4 (95% confidence interval = 3-7). Our study has shown that patients with HDs in tertiary care who opted for treatment with acupuncture appeared to receive similar clinical benefits to those that chose PDT, suggesting these treatments may be similarly effective of the prevention of headache in a real-world clinical setting.

Treatment of Hereditary Angioedema.

Hereditary angioedema due to C1-esterase inhibitor deficiency (C1-INH-HAE) is a rare autosomal dominant disease. In the last decade, new drugs and new indications for old drugs have played a role in the management of C1-INH-HAE. This review examines current therapy for C1-INH-HAE and provides a brief summary of drugs that are under development. Increased knowledge of the pathophysiology of C1-INH-HAE has been crucial for advances in the field, with inhibition of the kallikrein-kinin system (plasma kallikrein, activated factor XII) as a key area in the discovery of new drugs, some of which are already marketed for treatment of C1-INH-HAE. Pharmacological treatment is based on 3 pillars: treatment of acute angioedema attacks (on-demand treatment), short-term (preprocedure) prophylaxis, and long-term prophylaxis. The 4 drugs that are currently available for the treatment of acute angioedema attacks (purified plasma-derived human C1 esterase inhibitor concentrate, icatibant acetate, ecallantide, recombinant human C1 esterase inhibitor) are all authorized for self-administration, except ecallantide. Purified plasma-derived human C1 esterase inhibitor concentrate is the treatment of choice for short-term prophylaxis. Tranexamic acid, danazol, intravenous and subcutaneous nanofiltered purified plasma-derived human C1 esterase inhibitor concentrate, and lanadelumab can be used for long-term prophylaxis. New drugs are being investigated, mainly as long-term prophylaxis, and are aimed at blocking the kallikrein-kinin system by means of antiprekallikrein, antikallikrein, and anti-activated FXII action.

Survey of actual conditions of erythema marginatum as a prodromal symptom in Japanese patients with hereditary angioedema

BackgroundHereditary angioedema (HAE) is a rare but life-threatening condition. HAE types I and II (HAE-1/2) result from C1-inhibitor (C1–INH) deficiency. However, recent genetic analysis has established a new type of HAE with normal C1–INH (HAEnC1-INH). The mutations of factor XII, plasminogen, angiopoietin 1, and kininogen 1 genes may be the cause of HAEnC1-INH. Nevertheless, other causative molecules (HAE-unknown) may be involved. The Japanese therapeutic environment for HAE has been improving owing to the self-subcutaneous injection of icatibant, which was approved for the treatment of acute attack and enables early therapy. Erythema marginatum (EM) is a visible prodromal symptom which occasionally occurs prior to an angioedema attack; hence, recognizing the risk of an acute attack is important for early treatment. However, the detailed characteristics of EM remain unclear. In this study, we first investigated the clinical manifestations of EM in Japanese patients with HAE. MethodsA 20-point survey was developed and distributed to 40 physicians to gather clinical data on EM from patients with HAE. ResultsData on 68 patients with HAE (58 patients with HAE-1/2 and 10 patients with HAE-unknown) were collected. Of the patients with HAE-1/2, 53.4% experienced EM, whereas 43.1% did not. The forearm was the most frequent area of EM (64.5%), followed by the abdomen (29.0%) and upper arm and precordium (19.3%). Of the HAE-1/2 patients with EM, 41.9% always had angioedema following EM, while 29.0% always had colocalization of EM with angioedema. Moreover, 3.2% showed a correlation between the awareness of EM and severity of an angioedema attack. In 60.9% of HAE-1/2 patients with EM, the interval between the awareness of EM and appearance of angioedema was <3 h. Of the patients with HAE-unknown, 30.0% also experienced EM. ConclusionWe confirmed that more than one-half of Japanese patients with HAE-1/2 and one-third of those with HAE-unknown develop EM as the prodromal symptom of an angioedema attack. Physicians should communicate the significance of EM to patients with HAE to prepare them for possible imminent attacks.

Health technology assessment for the acute and preventive treatment of migraine: A position statement of the International Headache Society.

The Clinical Trials Subcommittee of the International Headache Society presents the first Health Technology Assessment for the Acute Treatment of Migraine Attacks and Prevention of Migraine. Health technology assessments are systematic evaluations of the properties, effects, and consequences of healthcare technologies; this position statement is designed to inform decision makers about access to and reimbursement for medications and devices for the acute and preventive treatment of migraine. This position statement extends beyond the already available guidelines on randomized controlled trials for migraine to incorporate real-world evidence and a synthetic approach for considering multiple data sources and modelling methods when assessing the value of migraine treatments.

Acquired Angioedema with C1 Inhibitor Deficiency: Occurrence, Clinical Features, and Management: A Nationwide Retrospective Study in the Czech Republic Patients

Introduction: Acquired angioedema with C1 inhibitor deficiency (AAE-C1-INH) is rare but a potentially life-threatening disease. There are no official prevalence data, nor approved therapies for this condition. Objective: In this study, we aimed to collect and analyze clinical data on patients with AAE-C1-INH in the Czech Republic. Methods: We have conducted a retrospective analysis of AAE-C1-INH patients from Czech referral centers for the treatment of hereditary angioedema with C1 inhibitor deficiency. The inclusion criteria involved recurrent episodes of angioedema with the first manifestation at or after the age of 40, negative family history of angioedema, and C1 inhibitor function 50% or less. Results: A total of 14 patients (7 males and 7 females) met the inclusion criteria for AAE-C1-INH. The median age of the symptom onset was 59.5 years, and the median diagnosis delay was 1 year. The most common clinical manifestation was facial edema (100%) and upper airway swelling (85.7%). All patients responded to the acute attack treatment with icatibant and plasma-derived or recombinant C1 inhibitor concentrate. Lymphoid malignancy was identified in 9 patients (64%), monoclonal gammopathy of uncertain significance in 3 (21%), and in 1 patient autoimmune disease (ulcerative colitis) was considered causative (7%). We were not able to identify any underlying disease only in 1 patient (7%). In 6 of 7 patients (86%) treated for lymphoma, either a reduction in the frequency of angioedema attacks or both angioedema symptoms’ disappearance and complement parameter normalization was observed. Conclusions: The prevalence of AAE-C1-INH in the Czech Republic is about 1:760,000. This rare condition occurs in approximately 8% of the patients with angioedema with C1 inhibitor deficiency. AAE-C1-INH is strongly associated with lymphoproliferative disorders, and treating these conditions may improve the control of angioedema symptoms.

Formulation and stability study of hydroxychloroquine sulfate oral suspensions

Hydroxychloroquine is an antimalarial drug indicated in the treatment of acute attacks of malaria due to Plasmodium vivax, P. malariae, P. ovale, and susceptible strains of P. falciparum. It is also used for the treatment of rheumatoid arthritis, discoid and systemic lupus erythematosus, and more recently proposed in COVID-19 therapy. Hydroxychloroquine is only available in tablets which are not easy to administer for pediatric and geriatric patients, and patients unable to swallow such as patients found in intensive care units. The aim of this work was to develop and optimize a ready to use liquid hydroxychloroquine formulation and to carry out the corresponding chemical and microbiological stability studies. The formulation was evaluated for ease of preparation, physical properties, and palatability. Its stability was performed at ambient temperature and under refrigeration. After 6 months of stability testing, the results showed no pH change, no drug loss, no microbial development, and no visual change. The formulation, employing excipients in a range that EMA has recommended, showed chemical and microbiological stability for at least 6 months even in the worst storage conditions.

Salbutamol-induced lactic acidosis in status asthmaticus survivor

BackgroundSalbutamol-induced lactic acidosis is a rare presentation that could manifest in specific clinical context as acute asthmatic attack treatment. An increase of glycolysis pathway leading to pyruvate escalation is the mechanism of hyperlactatemia in β2-adrenergic agonist drug.Case presentationA 40-year-old man who had poor-controlled asthma, presented with progressive dyspnea with coryza symptom for 6 days. He was intubated and admitted into medical intensive care unit due to deteriorated respiratory symptom. Severe asthmatic attack was diagnosed and approximate 1.5 canisters of salbutamol inhaler was administrated within 24 h of admission. Initial severe acidosis consisted of acute respiratory acidosis from ventilation-perfusion mismatch and acute metabolic acidosis resulting from bronchospasm and hypoxia-related lactic acidosis, respectively. The lactate level was normalized in 6 h after hypoxemia and ventilation correction. Given the lactate level re-elevated into a peak of 4.6 mmol/L without signs of tissue hypoxia nor other possible etiologies, the salbutamol toxicity was suspected and the inhaler was discontinued that contributed to rapid lactate clearance. The patient was safely discharged on the 6th day of admission.ConclusionThe re-elevation of serum lactate in status asthmaticus patient who had been administrated with the vast amount of β2-adrenergic agonist should be considered for salbutamol-induced lactic acidosis and promptly discontinued especially when there were no common potentials.

Comparison of the efficacy of nebulized budesonide and systemic steroids in children admitted to the emergency service with acute asthma attacks

Aim: Systemic steroids are used when necessary in the treatment of acute asthma attacks, but they have a high potential for side effects. The role of nebulized steroids in acute asthma attacks is controversial and there are few studies on the subject. In our study, it was aimed to evaluate the efficacy of nebulized budesonide treatment in children presenting with acute asthma attack.Materials and Methods: In this study, patients aged 5-18 years who presented to emergency service with a mild acute asthma attack were evaluated. Patients with mild acute asthma attack who did not respond to nebulized salbutamol treatment at 3 doses (0.15 mg/kg/dose) administered within the first hour, were included in the study. The patients were randomly divided into two groups. Group A received single-dose systemic steroid (1mg/kg) while Group B received single-dose nebulized budesonide (500µg). The groups were compared in terms of demographics, % O2 saturation, pulmonary index score (PIS), and duration of hospital stay. Results: A total of 61 cases were enrolled to the study (Group A n=33, Group B n=28). There was no statistically significant difference between groups in terms of mean age and gender (P>0.05). In both groups, % O2 saturation values at 4th hour of treatment increased and PIS regressed significantly compared to the baseline values (P0.001). It was also found that the % O2 saturation values at 4th hour were higher in patients treated with nebulized budesonide compared to patients treated with systemic steroids (p:0.02). There was no statistically significant difference between the groups in terms of duration of hospital stay (P>0.05). Conclusion: Based on the results of our study, a single dose (500µg) of nebulized budesonide was shown to be as effective as systemic steroid therapy in the treatment of mild acute asthma attacks.

Pharmacological and clinical study results of Berotralstat Hydrochloride for long-term prophylactic treatment of hereditary angioedema

Hereditary angioedema (HAE) is a rare disease that causes serious health problem and affects on quality of life for patient due to recurrent episodes of angioedema in various body such as the skin, larynx, digestive tract, and limbs. Many HAE patients have deficiency or dysfunction of C1 inhibitor, impaired regulation of plasma kallikrein activity and overproduction of bradykinin, resulting in leading to episodes of increased capillary hyper permeability and angioedema. Therapy of HAE consists of on-demand treatment for acute attack and prophylactic treatment by suppressing the onset of acute attack in the short and long term. However, no drug has been approved for long-term prophylaxis in Japan. Berotralstat hydrochloride (ORLADEYO Capsules 150 mg) is an oral, selective plasma kallikrein inhibitor approved for the suppression of the onset of acute attacks in HAE in Japan in January 2021. Preclinical studies demonstrated that Berotralstat is a potent and highly specific inhibitor of human plasma kallikrein activity. Berotralstat suppressed bradykinin production in the HUVEC system. Clinical studies demonstrated that oral administration of Berotralstat to HAE type I or type II patients at a dose of 150 mg once daily showed a reduction of HAE attack rate and clinically significant change in angioedema quality of life score. The most common side effect was gastrointestinal symptoms. In conclusion, preclinical and clinical data indicated that Berotralstat is an effective treatment for long-term prophylactic treatment by suppressing the onset of acute attack in HAE patient and is considered to be a useful treatment option for patients.

Hereditary Angioedema: Diagnostic Algorithm and Current Treatment Concepts.

Hereditary angioedema (HAE) is an uncommon disorder with a global prevalence of approximately 1 in 10,000 to 1 in 50,000 population. This disease is grossly underrecognized in India because of lack of awareness and/or lack of diagnostic facilities. Clinical manifestations include swelling over face, eyes, lips, hands, feet, and genitals, abdominal pain, and life-threatening laryngeal edema. HAE should be suspected in all patients who present with angioedema without wheals and who do not respond to antihistamines and/or steroids. C1 levels, C1-INH levels, and C1-INH function should be checked in all patients suspected to have HAE. C1q levels should be assessed in patients with suspected autoimmune-mediated acquired angioedema. Management of HAE constitutes the treatment of acute attack and short-term and long-term prophylaxis. Because of lack of all first-line recommended medications, the management of HAE in India is a challenging task. Patients are managed using fresh frozen plasma (acute treatment), tranexamic acid, and attenuated androgens (prophylaxis). Even though attenuated androgens have been shown to be effective in the prevention of attacks of HAE, the side effect profile especially in children and in females is a serious concern. Hence, the treatment needs to be individualized considering the risk-benefit ratio of long-term prophylaxis. In this review, we provide an overview of diagnostic strategy for patients with HAE and the current treatment concepts with emphasis on currently available treatment options in resource-constrained settings.

Type 2 hereditary angioedema treated with acute exacerbation with icatibant: A case report

Hereditary angioedema (HAE) is a rare autosomal dominant disorder caused by genetic deficiency or decreased function of C1-es terase inhibitor. It is characterized by swelling of subcutaneous and submucosal tissues of the extremities, gastrointestinal tract, and upper airways which can be life-threatening. Thus, early recognition and treatment of the disease are important. Short- and long-term prophylaxes are used to decrease the severity and frequency of attacks. Icatibant is a selective bradykinin B2-receptor antago nist, earlier treatment of acute attacks and hospital admission. The authors present a case of 47-year-old woman who was diag nosed with type II HAE, danazol as a long term. Her symptoms improved dramatically after drug treatment. The unpredictability and recurrence of HAE attacks could have a negative impact on social life and quality of life. This case shows that timely and proper treat ment could improve quality of life and reduce morbidity and mortality. (Allergy Asthma Respir Dis 2021;9:180-183)

Bronchodilatory effects of B-type natriuretic peptide in acute asthma attacks: a randomized controlled clinical trial.

B-type natriuretic peptide (BNP) regulates different physiological processes such as blood pressure, cardiac growth, and neural and skeletal development. Thus, the aim of this study w as to evaluate the effect of BNP in the treatment of acute asthma attacks. In this randomized clinical trial, patients with acute asthma attacks were enrolled. The patients were divided randomly into two groups. Patients in the interventional group received BNP via intravenous infusion. Two µg/kg of BNP was injected as a bolus in 60 seconds. Then, infusion of BNP immediately began and was given in 0.01, 0.02, and 0.03 µg/kg/min doses every 30 minutes for the first 1.5 hours. The patients in the control group received nebulized salbutamol. Afterwards, peak flow meter findings, hemodynamic parameters, and estimation of the clinical severity of asthma in both groups were checked every 30 minutes. In total, 40 patients were included in this study. The values of PEFR in the 60th and 90th minutes in the control group were lower than those in the interventional group. In the 60th minute, the mean of PEFR was 377.3 in the BNP group but 335.95 in the control group (P = 0.049). Moreover, this difference remained significant in the 90th minute (P = 0.021). However, forced expiratory volume in one second (FEV1) did not differ between the groups at any time (p > 0.05). Although a large experimental study is needed to verify our hypothesis, it seems that BNP might be a therapeutic option in asthma exacerbations, particularly in those with b2 agonist receptor polymorphism.

Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease: Current Insights into the Disease Pathophysiology, Diagnosis and Management.

Myelin oligodendrocyte glycoprotein (MOG)-associated disease (MOGAD) is a rare, antibody-mediated inflammatory demyelinating disorder of the central nervous system (CNS) with various phenotypes starting from optic neuritis, via transverse myelitis to acute demyelinating encephalomyelitis (ADEM) and cortical encephalitis. Even though sometimes the clinical picture of this condition is similar to the presentation of neuromyelitis optica spectrum disorder (NMOSD), most experts consider MOGAD as a distinct entity with different immune system pathology. MOG is a molecule detected on the outer membrane of myelin sheaths and expressed primarily within the brain, spinal cord and also the optic nerves. Its function is not fully understood but this glycoprotein may act as a cell surface receptor or cell adhesion molecule. The specific outmost location of myelin makes it a potential target for autoimmune antibodies and cell-mediated responses in demyelinating processes. Optic neuritis seems to be the most frequent presenting phenotype in adults and ADEM in children. In adults, the disease course is multiphasic and subsequent relapses increase disability. In children ADEM usually presents as a one-time incident. Luckily, acute immunotherapy is very effective and severe disability (ambulatory and visual) is less frequent than in NMOSD. A critical element of reliable diagnosis is detection of pathogenic serum antibodies MOG with accurate, specific and sensitive methods, preferably with optimized cell-based assay (CBA). MRI imaging can also help in differentiating MOGAD from other neuro-inflammatory disorders. Reports on randomised control trials are limited, but observational open-label experience suggests a role for high-dose steroids and plasma exchange in the treatment of acute attacks, and for immunosuppressive therapies, such as steroids, oral immunosuppressants and rituximab as maintenance treatment. In this review, we present up-to-date clinical, immunological, radiographic, histopathological data concerning MOGAD and summarize the practical aspects of diagnosing and managing patients with this disease.

Anti-MOG associated disorder–Clinical and radiological characteristics compared to AQP4-IgG+ NMOSD–A single-center experience

BackgroundThe discovery of two immunoglobulin G (IgG) antibodies against aquaporin 4 (anti-AQP4) and myelin oligodendrocyte glycoprotein (anti-MOG) has led to the distinction of the disorders anti-AQP4 immunoglobulin G positive neuromyelitis spectrum disorder (AQP4-IgG+ NMOSD) and anti-MOG associated disorder (MOGAD). Different clinical and radiological features have been proposed to distinguish these two demyelinating CNS diseases. MethodsThis is a single-center retrospective review at the University of Florida (UF) including all patients with the diagnostic code ICD G36 (“other acute disseminated demyelination”) from October 2015 to January 2020 (n=141) and all charts included in the clinical NMOSD database of the UF Division of Neuroimmunology (n=36). A total of 151 cases were reviewed for presence of anti-MOG and anti-AQP4 antibodies and NMOSD diagnostic criteria. Differences in MOGAD and AQP4-IgG+ NMOSD were compared. ResultsOf the 151 reviewed patient charts, 11 were consistent with MOGAD and 43 with AQP4-IgG+ NMOSD. Patients with MOGAD were significantly younger at symptom onset compared to patients with AQP4-IgG+ NMOSD (14 [1-33] years vs. 37 [6-82] years; p=0.005).In comparison with AQP4-IgG+ NMOSD, optic neuritis in MOGAD was more frequently associated with bilateral optic nerve involvement: (6/11 [54.5%] vs. 6/43 [13.9%]; p=0.009) and fundoscopic presence of optic disc edema (5/11 [45.5%] vs. 3/43 [7%]; p=0.006). Perineuritis was a common radiological feature in MOGAD (present in 4 cases). In case of myelitis, there was more frequent involvement of the conus medullaris in MOGAD (4/11 [36.4%] vs. 2/43 [4.7%]; p=0.012). Symptomatic cerebral syndrome with supratentorial white matter lesions was seen in MOGAD patients with pediatric onset (pediatric onset: 4/6 [66.7%] vs. adult onset: 0/5 [0%]. In MOGAD, evidence for combined central and peripheral demyelination and increased intracranial pressure was present in one patient each. A preceding inciting event (illness/postpartum) was more frequently identifiable in MOGAD (4/11 [36.4%] vs. 4/43 [7%]; p=0.045).Disability as calculated on the Expanded Disability Status Scale was less severe in MOGAD compared to AQP-IgG+ NMOSD (most severe presentation: 5 [2-7] vs. 7 [1-10]; p=0.015; most recent assessment: 2 [0-5] vs. 5 [0-10]; p=0.045) and patients were more likely to respond to treatment of acute attacks with corticosteroids and/or plasmapheresis (Clinical Global Impression-Global Change scale: 1 [1-4] vs. 3 [1-6]; p=0.001). InterpretationThe study confirms that simultaneous bilateral optic neuritis, presence of optic disc edema, transverse myelitis with conus involvement and a less severe disease course are distinctive features of MOGAD.

Experience sharing on treatment of an acute colchicine poisoning patient

Colchicine is mainly used in the treatment of acute gout attack, acute attack of chronic gout and other diseases in clinic. But the drug has high toxicity (highly toxic), the therapeutic dose is very close to the toxic dose, and there is no special effect after poisoning drug detoxification. The clinical manifestations of early patients after poisoning are not specific, which brings some difficulties and challenges in clinical diagnosis and treatment, and virtually increases the probability of death from drug poisoning. The drug poisoning is still sporadic reports. On September 1, 2020, a patient with acute colchicine poisoning was admitted to People's Hospital of Songtao Miao Autonomous County. This patient is the one with the longest poisoning time that can be retrieved by authoritative medical journals. After treatment, the patient was cured and discharged. This article reported the experience of diagnosis and treatment.

P164 POPULATION PHARMACOKINETICS OF RECOMBINANT HUMAN C1 ESTERASE INHIBITOR (RHC1-INH) IN CHILDREN WITH HEREDITARY ANGIOEDEMA (HAE)

rhC1-INH is indicated in the US for the treatment of acute HAE attacks in adolescents/adults; it is also indicated for children aged ≥2 years in the EU. There is a need to gain further insight into pharmacokinetic (PK) differences in functional C1-INH levels by age (ie, children, adolescents, and adults). The current aim was to employ population PK modeling to predict C1-INH levels following rhC1-INH administration.