rescue medication
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Author(s):  
Umar Afzal ◽  
Abdul Nasir Ansari ◽  
Mohd Nayab

Shaqīqa-i-Muzmin (chronic migraine) is a type of migraine that is usually caused by cold humours (bārid akhlāṭ), specifically by phlegm (balgham) and to lesser extent by black bile (sauda). The aim of this study was to evaluate the effect of Nutul-i-Ḥār (hot irrigation) in the treatment of chronic migraine. Nutul (irrigation) therapy is widely and successfully used in diseases of head as described in Unānī system of medicine. This open, single-arm, exploratory clinical study was conducted in 30 patients of chronic migraine selected by convenient sampling method. One litre decoction prepared with 12 g each of Astragalus hamosus L. (Iklilul malik), Matricaria chamomilla L. (Babuna), Artemisia absinthium L. (Afsanteen), Origanum vulgare L. (Marznjosh) and Trigonella foenum graecum L. (Hulba) was poured over painful side of head for 45 minutes on every alternate day for a period of 30 days (15 sittings). The patients, thereafter, were followed untill 90th day of the study for various outcome measures comprising headache intensity, headache frequency, Migraine Disability Assessment Scale (MIDAS), and rescue medication. The reduction in headache frequency, MIDAS score and use of rescue medication was significant (p < 0.001) after the treatment. Reduction in headache intensity at 30th day, 60th day and 90th day was significant as compared with baseline values (p < 0.001) but not statistically significant at 90th day with respect to 60th day (p > 0.05). Statistical analysis was done using parametric (paired t-test) and non-parametric tests (Wilcoxan sign ranked test, Freidman with Dunn’s multiple comparison tests). Hot irrigation with medicated decoction was found effective in the treatment of chronic migraine.


2022 ◽  
Vol 16 ◽  
pp. 175346662110660
Author(s):  
Yiwen Gong ◽  
Yinghua Lv ◽  
Hongxia Liu ◽  
Qingshan Zheng ◽  
Lujin Li

Objective: This study aimed to quantitatively compare the efficacy and safety of long-acting β2-agonist (LABA)/long-acting muscarinic antagonist (LAMA) fixed-dose combinations (FDCs) for the treatment of stable chronic obstructive pulmonary disease (COPD), especially in terms of their loss of efficacy in lung function. Methods: Randomized controlled clinical trials of LABA/LAMA FDCs for the treatment of stable COPD were comprehensively searched for in public databases. Pharmacodynamic models were established to describe the time course of the primary outcome [trough forced expiratory volume in the first second (FEV1)]. Secondary outcomes [COPD exacerbations, St. George’s Respiratory Questionnaire (SGRQ), Transition Dyspnoea Index (TDI), and rescue medication use] and safety outcomes [mortality, serious adverse events (SAEs), and withdrawals due to adverse events (AEs)] were also compared via a meta-analysis. Results: A total of 22 studies involving 16,486 participants were included in this study. The results showed that in terms of primary outcome (change from baseline in trough FEV1), the efficacy of vilanterol/umeclidinium was the highest, while the efficacy of formoterol/aclidinium was the lowest, with a maximum effect value (Emax) of 0.185 L [95% confidence interval (CI): 0.173–0.197 L] and 0.119 L (95% CI: 0.103–0.135 L), respectively. The efficacy of other drugs, such as formoterol/glycopyrronium, indacaterol/glycopyrronium, and olodaterol/tiotropium, were comparable, and their Emax values were 0.150–0.177 L. Except for vilanterol/umeclidinium, the other four LABA/LAMA FDCs showed a certain degree of loss of efficacy. Compared with the efficacy at 2 days, the trough FEV1 (L) relative to baseline at 24 weeks decreased by 0.029–0.041 L. In terms of secondary outcomes, the efficacy of different LABA/LAMA FDCs was similar in TDI and rescue medication use. However, formoterol/aclidinium was better in preventing the COPD exacerbations, while vilanterol/umeclidinium was the best in terms of SGRQ. In addition, different LABA/LAMA FDCs and placebo had similar safety outcomes. Conclusion: The present findings may provide necessary quantitative information for COPD medication guidelines.


Author(s):  
Steven Alan Rice ◽  
Ruth Melinda Müller ◽  
Sarah Jeschke ◽  
Birthe Herziger ◽  
Thilo Bertsche ◽  
...  

AbstractFebrile seizures (FS) in children are common, but little is known about parents’ perceptions and knowledge of FS. We interviewed parents of children aged 6 months to 6 years affected by FS (FS group, 65 parents) or unaffected (control group, 54 parents). In the FS group, 32% said they knew their child had an FS when the first event occurred, and 89% described fear when the child had a seizure, with a median intensity of 10/10 (Q25/Q75: 9/10). Related to follow-up, 77% in the FS group (will) observe their child more carefully after the first seizure happened, and 63% (will) give antipyretics earlier at a median temperature of 38.2 °C (100.8 °F). In the FS group, 62% were unaware of FS before the first event (54% of control group did not know about FS thus far, n.s.). In the FS group, 20% would put a solid object in the mouth of a child having a seizure (control group, 39%, p = 0.030), and 92% would administer an available anti-seizure rescue medication (control group, 78%, p = 0.019). In the FS group, 71% feared that children with FS might suffocate (control group, 70%, n.s.).Conclusion: Information about FS and their management should be more available to improve parents’ coping and patient safety. What is Known:• Febrile seizures in children are common.• The prognosis of children suffering from febrile seizures is usually rather good. What is New:• Over half of parents had not informed themselves about febrile seizures so far; and only 32% of parents realized their child had a febrile seizure when it occurred.• Most parents described own fear with a median intensity of 10/10; and 63% (will) give antipyretics earlier at a median temperature of 38.2 °C (100.8 °F).


2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Lukas Israel ◽  
Gabriele Rotter ◽  
Ulrike Förster-Ruhrmann ◽  
Josef Hummelsberger ◽  
Rainer Nögel ◽  
...  

Abstract Background Acupuncture has shown beneficial effects for seasonal allergic rhinitis (SAR); however, it is time and cost intensive. We investigated feasibility and effects of self-administered body acupressure as a self-care technique that stimulates acupuncture points with manual pressure in SAR patients. Methods We conducted a two-armed randomized controlled exploratory trial to compare effects of self-administered acupressure over 4 weeks at five acupuncture points plus rescue medication (RM) with cetirizine compared to RM alone in SAR patients. Among other outcome parameters, we assessed disease-related quality of life (Rhinitis Quality of Life Questionnaire [RQLQ]), overall SAR symptoms by a visual analogue scale (VAS) and a rescue medication score (RMS) after 4 and 8 weeks. Results Forty-one SAR patients (mean age 38.5 ± 10.0 years, n = 21, 51.2% women) were randomized. Compared to RM alone (n = 21), acupressure plus RM (n = 20) was associated with relevant improvements after 4 weeks, shown by the difference between groups in adjusted means of RQLQ: − 0.9 points (95% CI − 1.6 to − 0.2; p = 0.011) and VAS overall SAR symptoms: − 21.6 mm (95% CI − 36.3 to − 6.8; p = 0.005). The RMS was lower in the acupressure group than in the control group: 1.9 points (95% CI − 3.8 to − 0.1; p = 0.120). Group differences decreased slightly until week 8. The acupressure was feasible and safe. Conclusion Results of this exploratory study indicate that self-applied acupressure is feasible, may improve disease-specific quality of life and reduce disease-related symptoms as well as anti-allergic medication intake in SAR patients. High-quality confirmatory studies including a sham-control group are needed in the future. Trial registration DRKS-ID: DRKS00014310. Date of registration in DRKS: 2018/04/24. Investigator sponsored/initiated trial (IST/IIT): yes. Ethics approval/approval of the ethics committee: Approved (leading) Ethics Committee No. EA1/033/18, Ethik-Kommission der Charité -Universitätsmedizin Berlin. URL: https://www.drks.de/drks_web/navigate.do?navigationId=trial.HTML&TRIAL_ID=DRKS00014310


2021 ◽  
Vol 10 (23) ◽  
pp. 5609
Author(s):  
Katarzyna Piotrowicz-Wójcik ◽  
Małgorzata Bulanda ◽  
Aldona Juchacz ◽  
Joanna Jamróz-Brzeska ◽  
Jacek Gocki ◽  
...  

Hereditary angioedema (HAE) due to C1-inhibitor (C1-INH) deficiency is a rare disease characterized by recurrent swellings. This study aims to determine (i) the clinical characteristics of the HAE patient population from Poland, and (ii) real-life patients’ treatment practices. A cross-sectional study involved 138 adult HAE patients (88 females, 50 males) treated in six regional HAE centers in Poland. Consecutive patients during routine follow-up visits underwent a structured medical interview on the clinical characteristics of the course and treatment of HAE attacks within the last six months. A total of 118 of 138 patients was symptomatic. They reported in total 2835 HAE attacks predominantly peripheral and abdominal, treated with plasma-derived C1-INH (61.4%), icatibant (36.7%) and recombinant C1-INH (1.9%). An amount of 116 patients carried the rescue medication with them while traveling, and 74 patients self-administrated on demand treatment. There were twice as many symptomatic women (n = 78) as there were men (n = 40). Women treated their HAE attacks significantly more often than men. Older patients (≥65 years) reported a longer delay in diagnosis, and practiced the self-administration of rescue medication less frequently in comparison to other patients. Clinical features of the surveyed population are similar to other European, but not Asian, HAE patient groups. Self-administration still remains an unmet medical need. Some distinct HAE patients may require special attention due to the severe course of the disease (females) or a delay in diagnosis (the elderly).


Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4243-4243
Author(s):  
Judith A. Boice ◽  
Tyler W. Buckner ◽  
Stacy E. Croteau ◽  
Nathaniel Katz ◽  
Robert F. Sidonio ◽  
...  

Abstract Background: Intra-articular bleeding (hemarthrosis) accounts for 80-90% (Roosendaal, et. al. Semin Thromb Hemost . 2003;29:37) of all bleeds and more than 90% of serious bleeding events in patients with severe hemophilia (Valentino. J Thromb Haemost . 2010;8:1895). Recurrent hemarthroses result in progressive joint damage and the development of hemophilic arthropathy (HA) in up to 50% of adults with hemophilia (Forsyth, et al. Haemophilia . 2014;20:44). Acetaminophen is commonly prescribed for pain due to HA yet has limited efficacy at therapeutic doses (Rodriguez-Merchan. Blood Rev. 2018;32:116). Traditional non-steroidal anti-inflammatory drugs (tNSAIDs) inhibit platelet function and cause gastrointestinal (GI) complications including bleeding, both of which can be harmful in patients with hemophilia (Rodriguez-Merchan. Blood Rev. 2018;32:116; Brooks, et al. Rheumatology . 1999;38(8):779). Opioids are prescribed in over 60% of patients with HA(Witkop, et al. Haemophilia. 2012;18:e115) despite dependence, potential for abuse, and an increased risk of falls and other opioid-related injury (Rodriguez-Merchan. Blood Rev. 2018;32:116). Given an unmet need in pain management for HA, alternate approaches are needed. TRM-201 (rofecoxib) is a cyclooxygenase-2 (COX-2) selective NSAID with no impact on platelet function and a lower GI risk than tNSAIDs (Vioxx (rofecoxib) package insert. Merck & Co. I, ed. Whitehouse Station, NJ, 2004). The RESET-HA study is designed to evaluate the efficacy and safety of rofecoxib for HA pain management. Methods/Design: RESET-HA is a multi-national, randomized, double-blind study to evaluate the efficacy and safety of rofecoxib in hemophilia A or B patients with diagnosed HA, aged 12 to 75 years and is based on a previous pilot study (Tsoukas, et al. Blood, 2006;107:1785). Patients must have a history of joint bleeding, and chronic symptomatic pain in one or more joint(s) on 20 of the 30 days prior to screening. Exclusion criteria include use of opioids for greater than 4 days/week, or opioid transdermal patches in the 30 days prior to screening, history of GI perforation, ulcer or bleeding, peptic ulcer disease and major cardiac ischemic symptoms or events. Randomized patients (n=80 per arm) are washed out of analgesics prior to daily administration of TRM-201 (17.5 mg/day) or placebo for 12 weeks (Part I) during which acetaminophen (Stage-1) and acetaminophen plus codeine (Stage-2) are available as rescue medication (where available and acceptable to the patient and study doctor). The patient assessment of hemophilic arthropathy pain, a 0- to 10-point numeric rating scale validated for the assessment of pain across many disease states, is recorded daily. The primary endpoint is the placebo adjusted change from baseline in weekly average of patient assessment of daily HA pain score at Week 12. Key secondary endpoints include patient assessments using the PROMIS physical function instrument and pain interference from the Brief Pain Inventory. Sleep disturbance is also measured using the PROMIS instrument. The study explores the efficacy of rofecoxib on the number of suspected joint bleeds and on the amount of rescue medication used. Following Part I, all study patients (regardless of original randomization arm) receive rofecoxib 17.5 mg once daily for up to 12 additional months (Part II). During Part II of the study, only acetaminophen will be provided as rescue medication. Discussion: The RESET-HA study is intended to evaluate the efficacy and safety of TRM-201 in patients with HA and is the first Phase III trial ever conducted to assess a treatment for HA pain. Pain management in HA requires analgesic anti-inflammatory treatment that does not exacerbate bleeding. Rofecoxib has been shown to have no effect on platelet function, even at supratherapeutic doses(Vioxx (rofecoxib) package insert. Merck & Co. Whitehouse Station, NJ, 2004), a decreased risk of GI side effects, including GI bleeding compared with tNSAIDs, and no greater cardiovascular risk than equipotent doses of COX-2 selective and tNSAIDs (U.S. Food and Drug Administration. J Pain Palliat Care Pharmacother. 2005;19:83). TRM-201 is anticipated to provide analgesic efficacy, with an acceptable tolerability and safety profile, and could become a new treatment option that may possibly facilitate the avoidance of opioid use in patients with HA. (NCT04684511) Disclosures Boice: Tremeau Pharmaceuticals Inc.: Current Employment, Current holder of individual stocks in a privately-held company, Current holder of stock options in a privately-held company. Buckner: Novo Nordisk: Honoraria; American Thrombosis: Membership on an entity's Board of Directors or advisory committees; CSL Behring: Honoraria; Genetech: Honoraria; Spark: Honoraria; Sanofi: Honoraria; Bayer: Honoraria; Pfizer: Honoraria; Takeda: Honoraria; Tremeau Pharmaceuticals: Consultancy, Honoraria; uniQure: Consultancy, Honoraria; BioMarin: Consultancy, Honoraria; Hemostasis Network: Membership on an entity's Board of Directors or advisory committees. Croteau: Tremeau Pharmaceuticals,Inc: Consultancy. Katz: Tremeau Pharmaceuticals,Inc: Consultancy. Sidonio: Takeda: Consultancy, Research Funding; Octapharma: Consultancy, Research Funding; Catalyst: Consultancy; Guardian Therapeutics: Consultancy; Bayer: Consultancy; Novo Nordisk: Consultancy; Biomarin: Consultancy; Pfizer: Consultancy; Genentech: Consultancy, Research Funding. Bolognese: Tremeau Pharmaceuticals,Inc: Consultancy. Garfield: Tremeau Pharmaceuticals,Inc: Current Employment, Current holder of individual stocks in a privately-held company, Current holder of stock options in a privately-held company. Corrigon: Tremeau Pharmaceuticals,Inc: Current Employment, Current holder of individual stocks in a privately-held company, Current holder of stock options in a privately-held company. Walsh: Genentech: Consultancy; Biomarin: Consultancy; Takeda: Consultancy; Novo Nordisk: Consultancy; Tremeau: Consultancy.


Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 1016-1016
Author(s):  
Vickie McDonald ◽  
Gerorge Loughlin ◽  
Andrew Laws ◽  
Duncan Stacey ◽  
Jeffrey Spears ◽  
...  

Abstract Immune thrombocytopenia (ITP) is a rare condition characterized by thrombocytopenia and variable bleeding severity. After acute management with steriods and intravenous immunoglobulin (IVIG), the next line therapies include medical treatments: immunosuppressive therapy such as rituximab, mycophenolate mofetil (MMF), thrombopoietin receptor agonists or surgical treatments. The choice of therapy depends on the time course of the disease, severity, patient choice and comorbidities. The evidence for comparative efficacy between different treatment modalities is lacking in the literature, in particular the immunosuppressive therapies MMF and rituximab. The UK ITP registry is a national registry of primary ITP, capturing demographics, clinical features, treatments and comorbidities. As of July 2021, it has 4402 participants from 90 acute NHS Trusts across the UK. We analyzed data from the UK adult ITP registry to assess responses to rituximab and MMF therapy. All patients entered into the registry from 1st January 2010 were included in this analysis. Internationally recognized definitions of response based on platelet count were used: partial response (PR) platelets &gt;30 x10 9/L and double baseline, complete response (CR) platelets &gt;100 x10 9/L; No response - absence of CR and PR. In addition, we reviewed use of rescue therapy and use of additional therapies following treatment. A total of 844 patients were included in the analysis, of these 486 had received rituximab and 358 had received MMF since 2010. The median follow up before treatment was 230.5 weeks for rituximab and 195 weeks for MMF. At the time of commencing rituximab (and within 4 weeks of starting), 26.1% patients were receiving prednisolone, 10% IVIG, 3% dexamethasone, 3.2% MMF, 2.95% romiplostim and 2.36% eltrombopag. At the time of commencing MMF (and within 4 weeks of starting), 39.2% patients were receiving prednisolone, 12.74 IVIG, 3.86% dexamethasone, 3.6 rituximab, 5.6% romiplostim and 3.86% eltrombopag. The total number of patients achieving platelet response at each time response are listed in table 1. Of those receiving rituximab with baseline platelets &lt;30 x10 9/L at starting therapy, 40% had at least PR by 4 weeks, 50% at 12 weeks and 30% at 12 months. For MMF, these percentages were 30%, 30% and 18% respectively, however, the numbers of patients in the MMF group were lower. The median duration of response was 44 weeks for rituximab and 27 weeks for MMF. Where new therapies were required, the median number of weeks until therapy was switched was 18 weeks for rituximab and 15 weeks for MMF. 77% patients and 80.7% patients who received rituximab and MMF respectively changed treatment during their follow up. Overall 284 (58.4%) patients received rescue medication during follow up, of those that received MMF 234 (65.36%) received rescue medication. The main rescue therapies were prednisolone (589 episodes in rituximab group and 520 in MMF group) and IVIG (482 in rituximab group and 321 in MMF group). In conclusion, this is one of the first studies, using real world data and long term follow up, to compare outcomes after immunosuppression. Both rituximab and MMF are often administered with additional therapies at the outset, predominantly steroids and IVIG, but also other therapies such as the thrombopoietin receptor agonists. The use of rescue therapies during follow up, suggesting treatment failure or a need to augment the response, is common with most patients in both groups ultimately having treatment change during follow up. Extension of this study to include other therapies and identifying groups most likely to respond will support optimization of treatment for patients with ITP. Figure 1 Figure 1. Disclosures McDonald: Bayer, Sobi, Novartis, Amgen, argenx: Honoraria; Grifols: Research Funding. Loughlin: FIECON: Current Employment. Laws: FIECON: Current Employment. Stacey: Grifols: Current Employment. Spears: Grifols: Current Employment. Hill: Apellis: Consultancy, Honoraria; Argenx: Consultancy; Novartis: Consultancy, Honoraria; Grifols: Consultancy, Honoraria; Sanofi: Consultancy; ReAlta: Consultancy; Alexion: Honoraria; Amgen: Honoraria. Cooper: UCB: Honoraria; Sanofi: Honoraria; Novartis: Honoraria, Other: Research support; Rigel: Honoraria, Other: Research support; Principia: Honoraria; Amgen: Honoraria; Grifols: Honoraria; Sobi: Honoraria. Makris: Freeline: Consultancy. Bradbury: BMS Pfizer: Honoraria, Speakers Bureau; Bayer: Honoraria, Other: support to attend conferences, Speakers Bureau; Amgen: Honoraria, Other: support to attend conferences, Speakers Bureau; Novartis: Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: support to attend conferences, Speakers Bureau; Lilly: Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Janssen: Honoraria, Speakers Bureau; Portola: Membership on an entity's Board of Directors or advisory committees; Ablynx: Membership on an entity's Board of Directors or advisory committees. Newland: Octapharma: Research Funding; Roche: Speakers Bureau; UCB Biosciences: Consultancy; Novartis: Consultancy, Research Funding, Speakers Bureau; Grifols: Consultancy, Speakers Bureau; GSK: Consultancy, Research Funding, Speakers Bureau; BMS: Research Funding; Argenx: Consultancy, Speakers Bureau; Angle: Consultancy; Amgen: Consultancy, Research Funding, Speakers Bureau. OffLabel Disclosure: rituximab - immune thrombocytopenia mycophenolate - immune thrombocytopenia


Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4210-4210
Author(s):  
Vickie McDonald ◽  
Axel Matzdorff ◽  
Åsa Mellbring ◽  
Jameel Nazir ◽  
Daniel Lindqvist ◽  
...  

Abstract Background: Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by low platelet counts caused by a combination of both impaired platelet production and increased peripheral platelet destruction. Current first-line ITP treatments include glucocorticoids and intravenous immunoglobulin (IVIG). However, these drugs have variable and transient efficacy, significant toxicities, and relapse is common upon discontinuation. Subsequent treatment options include the thrombopoietin receptor agonists (TPO-RAs). Avatrombopag (AVA) is an orally administered small molecule TPO-RA. It binds to the human TPO receptor (c-Mpl) at a site that is different from the endogenous TPO binding, stimulating signal transduction and mimicking the biological effects of endogenous TPO. In phase 2 and 3 studies in patients with ITP, AVA was administered to 128 patients for a median duration of 7 months (maximum duration &gt;2 years; Birhiray R, et al. EHA 2020). In a phase 3 trial (NCT01438840), the primary efficacy endpoint of cumulative number of weeks with platelet count ≥50×10 9/L during 6 months of treatment in the absence of rescue therapy was statistically significant favoring AVA over placebo. The most common treatment-emergent adverse events (AEs) in these phase 2 and 3 trials were headache, fatigue, and contusion. AVA has no significant hepatotoxicity and is administered with food without restrictions on meal composition. AVA is approved by FDA and EMA for the treatment of primary chronic ITP in adult patients who are refractory to other treatments (e.g. corticosteroids, IVIG). However, there is a need to provide data to treaters and the ITP community on the real-world usage and effectiveness of AVA (including patients previously treated with TPO-RAs). Described here is the rationale and design of the ADOPT study (NCT04943042), evaluating the use and effectiveness of AVA in adult patients with ITP in routine clinical practice in Europe. Study design and methods: This is a multicenter, observational, phase 4 study, with the primary objective to describe the real-world effectiveness of AVA treatment over a prospective period of 12 months in adult patients with ITP. Prospective data will be collected at routine clinical visits. In addition, retrospective data will be collected from patients' medical records for up to 12 months prior to AVA treatment start. Eligible patients must be ≥18 years, have provided informed consent, have an established and well documented ITP diagnosis, and are treated with or initiating treatment with AVA for ITP at enrollment. Decision to initiate treatment shall be made by the treating physician, independently from the decision to include the patient in the study. Exclusion criteria include enrollment in other clinical interventional study or intake of an investigational medicinal product ≤3 months prior to inclusion, and secondary ITP. The primary endpoint is cumulative number of weeks with a platelet count ≥30×10 9/L during AVA treatment. Platelet counts during rescue medication use and within 4 weeks after stopping a rescue medication or following splenectomy are considered non-response and thus not included in the primary endpoint cumulative number. Secondary endpoints supporting the primary objective include cumulative number of weeks with a platelet count ≥50×10 9/L, patients (n [%]) with a platelet count ≥30×10 9/L and ≥50×10 9/L for at least 8 consecutive weeks, patients (n [%]) experiencing WHO bleeding grade ≥2, patients (n [%]) requiring rescue medication, and time from AVA treatment start to platelet count ≥30×10 9/L and ≥50×10 9/L. Additional secondary endpoints are AVA dose and dosing frequency, reason for ITP treatment discontinuation or change from one ITP treatment to another (prior to and during the study), use of concomitant ITP medications, physician satisfaction with outcome of AVA treatment (5-point scale), physician assessment of clinical change of AVA treatment (Clinical Global Impression of Change scale), healthcare resource use, as well as a number of patient reported outcomes (Table 1). Secondary safety endpoints include serious AEs, AEs of special interest (thromboembolic events, significant bleeding) and AEs leading to AVA discontinuation. Data will be summarized using descriptive statistics. Study status: The study is planned to start 2021 and aims to enroll 150 patients. Figure 1 Figure 1. Disclosures McDonald: Grifols: Research Funding; Bayer, Sobi, Novartis, Amgen, argenx: Honoraria. Matzdorff: Grifols: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Amgen: Consultancy, Honoraria; Argenx: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; UCB: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Roche: Current holder of individual stocks in a privately-held company. Mellbring: Sobi: Current Employment. Nazir: Sobi: Current Employment. Lindqvist: Sobi: Current Employment. Santagostino: Sobi: Current Employment.


2021 ◽  
Vol 42 (6) ◽  
pp. 461-470 ◽  
Author(s):  
Michael Blaiss ◽  
William Berger ◽  
Bradley Chipps ◽  
Vivian Hernandez-Trujillo ◽  
Wanda Phipatanakul ◽  
...  

Background: Ciclesonide (CIC) is an inhaled corticosteroid (ICS) approved for the maintenance treatment of asthma in patients ages ≥ 12 years. The prodrug aspect of CIC is associated with a safety profile that may make it ideal for children. Objective: The objective was to summarize efficacy results from the eight phase III, randomized, double-blind, controlled trials in children with asthma conducted during CIC clinical development. Methods: Four trials compared CIC 40, 80, or 160 µg/day with placebo. Two trials compared CIC 160 µg/day with fluticasone propionate 200 µg/day, one trial compared CIC 80 or 160 µg/day with fluticasone 200 µg/day, and one trial compared CIC 160 µg/day with budesonide 400 µg/day. Results: The primary end point was met by at least two CIC doses versus placebo in the trials in which the primary end point was the change from baseline in lung function outcome (forced expiratory volume in 1 second [FEV1] % predicted or morning peak expiratory flow [PEF]). A trial that compared CIC with placebo did not meet the primary end point of superiority in time-to-first severe wheeze exacerbation or lack of improvement. The primary end point of noninferiority to the active control (fluticasone or budesonide) in the change from baseline in a lung function outcome (FEV1, morning PEF, evening PEF) was met with the CIC 160-µg dose in all active control trials. CIC generally demonstrated statistically significant improvements in forced expiratory flow at 25%‐75% of forced vital capacity, asthma symptoms, rescue medication use, and asthma control when compared with placebo and noninferiority for these outcomes compared with fluticasone or budesonide. Conclusion: In children with asthma, once-daily CIC significantly improved large and small airway function, asthma symptoms, and asthma control, and reduced rescue medication use compared with placebo. CIC was comparable with other ICS used to treat asthma in children, which demonstrated its worth for the pediatric population.


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