What can we expect for adolescents and adults with haemophilia switched to low‐dose prophylaxis from episodic treatment for over 3 years? A real‐world snapshot in China

Haemophilia ◽  
2021 ◽  
Author(s):  
Sha Liu ◽  
Ping‐yang Zhang ◽  
Zhi‐bin Jin ◽  
Yan‐hui Yuan ◽  
Min Wu ◽  
...  
Keyword(s):  
Real World ◽  
Low Dose ◽  
Episodic Treatment ◽  
Adolescents And Adults

scholarly journals Effects of replacement therapies with clotting factors in patients with hemophilia: A systematic review and meta-analysis

PLoS ONE ◽  
2022 ◽  
Vol 17 (1) ◽  
pp. e0262273
Author(s):  
Carolina J. Delgado-Flores ◽  
David García-Gomero ◽  
Stefany Salvador-Salvador ◽  
José Montes-Alvis ◽  
Celina Herrera-Cunti ◽  
...  
Keyword(s):  
Low Dose ◽  
Prophylactic Treatment ◽  
Hemophilia A ◽  
Clotting Factor ◽  
High Dose ◽  
Bleeding Rate ◽  
Episodic Treatment ◽  
Grade Methodology ◽  
Inform Decision Making ◽  
Intermediate Dose

Background Different prophylactic and episodic clotting factor treatments are used in the management of hemophilia. A summarize of the evidence is needed inform decision-making. Objective To compare the effects of factor replacement therapies in patients with hemophilia. Methods We performed a systematic search in PubMed, Central Cochrane Library, and Scopus. We included randomized controlled trials (RCTs) published up to December 2020, which compared different factor replacement therapies in patients with hemophilia. Random-effects meta-analyses were performed whenever possible. The certainty of the evidence was assessed using the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) methodology. The study protocol was registered in PROSPERO (CRD42021225857). Results Nine RCTs were included in this review, of which six compared episodic with prophylactic treatment, all of them performed in patients with hemophilia A. Pooled results showed that, compared to the episodic treatment group, the annualized bleeding rate was lower in the low-dose prophylactic group (ratio of means [RM]: 0.27, 95% CI: 0.17 to 0.43), intermediate-dose prophylactic group (RM: 0.15, 95% CI: 0.07 to 0.36), and high-dose prophylactic group (RM: 0.07, 95% CI: 0.04 to 0.13). With significant difference between these subgroups (p = 0.003, I2 = 82.9%). In addition, compared to the episodic treatment group, the annualized joint bleeding rate was lower in the low-dose prophylactic group (RM: 0.17, 95% CI: 0.06 to 0.43), intermediate-dose prophylactic group (RM of 0.14, 95% CI: 0.07 to 0.27), and high-dose prophylactic group (RM of 0.08, 95% CI: 0.04 to 0.16). Without significant subgroup differences. The certainty of the evidence was very low for all outcomes according to GRADE methodology. The other studies compared different types of clotting factor concentrates (CFCs), assessed pharmacokinetic prophylaxis, or compared different frequencies of medication administration. Conclusions Our results suggest that prophylactic treatment (at either low, intermediate, or high doses) is superior to episodic treatment for bleeding prevention. In patients with hemophilia A, the bleeding rate seems to have a dose-response effect. However, no study compared different doses of prophylactic treatment, and all results had a very low certainty of the evidence. Thus, future studies are needed to confirm these results and inform decision making.

31.8 REAL-WORLD SAFETY PROFILE OF DELAYED-RELEASE AND EXTENDED-RELEASE METHYLPHENIDATE FOR ADHD IN CHILDREN, ADOLESCENTS, AND ADULTS

2020 ◽  
Vol 59 (10) ◽  
pp. S213
Author(s):  
Ann Catherine Childress ◽  
Victor Otcheretko ◽  
Lewis E. Warrington ◽  
Justin A. Barnes ◽  
Ryan A. Gregg ◽  
...  
Keyword(s):  
Real World ◽  
Safety Profile ◽  
Extended Release ◽  
Delayed Release ◽  
Adolescents And Adults

scholarly journals Overall survival with oral selinexor plus low‐dose dexamethasone versus real‐world therapy in triple‐class‐refractory multiple myeloma

eJHaem ◽  
2020 ◽  
Author(s):  
Paul G. Richardson ◽  
Sundar Jagannath ◽  
Ajai Chari ◽  
Dan T. Vogl ◽  
Meletios A. Dimopoulos ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Overall Survival ◽  
Real World ◽  
Low Dose ◽  
Refractory Multiple Myeloma

scholarly journals P179 Glucocorticoid exposure and link to serious adverse events in patients with ANCA-associated vasculitis

Rheumatology ◽  
2020 ◽  
Vol 59 (Supplement_2) ◽  
Author(s):  
Philip Spearpoint ◽  
Cormac Sammon ◽  
Antonio Ramirez de ◽  
Arellano Serna ◽  
Peter Rutherford
Keyword(s):  
Cardiovascular Disease ◽  
Adverse Events ◽  
Renal Disease ◽  
Real World ◽  
Low Dose ◽  
Remission Induction ◽  
High Dose ◽  
Anca Associated Vasculitis ◽  
Increased Risk ◽  
New Onset

Abstract Background Remission induction in ANCA-associated vasculitis (AAV) is with high dose glucocorticoids (GC) and immunosuppressants. Patients are exposed to high GC dose and/or prolonged low dose. EULAR/EDTA guidelines target 7.5-10mg at 3 months but acknowledge this is often achieved later. This study used UK real world practice data to examine the scale of GC exposure and associated clinical risks in AAV. Methods The study utilised the Clinical Practice Research Datalink (CPRD) - Hospital Episode Statistics (HES) linked database. AAV patients were identified using specific READ and ICD codes and followed between 01/01/1997 and 01/01/2018. GP prescriptions were used to describe periods of continuous GC use, stop and restart and when high dose (> 30mg/day) and low dose (<30mg/day) was prescribed. Diagnostic codes indicative of infections and adverse events linked to GCs were used to estimate rates in the AAV population using a generalized linear model with a Poisson distribution. Results 450 AAV patients with at least one GC prescription were analysed. The median dose decreased to 9.3 mg (IQR 5.0 - 17.0) at 6 months and 5.1 mg (0.00 - 10.0) at 12 months,50% patients were taking > 10mg at 5 months and 25% were still > 10mg at 12 months. However, within 6 months of achieving 10mg/day, 50% relapse to needing dose >10mg, 75% within 2 years and 90% within 6 years. In adjusted Poisson model (age, gender, year of diagnosis before/after 2013) the rate of infection in AAV patients taking high dose was 2.59 times (CI95 1.95, 3.45) that of those on low dose and lower in those not taking GCs (IRR 0.27 (0.22-0.34)). Increased risk of new onset cardiovascular disease (IRR 2.55 (0.92, 7.04)) and new onset renal disease (IRR 3.4 (1.29-8.96)) were higher in patients receiving high dose. Conclusion AAV patients have significant exposure to high dose GCs and in real world practice, GC dose remains higher than recommended in current clinical guidelines. High dose GCs are associated with high risk of infection and new cardiovascular disease and renal disease. This creates a significant patient burden and has implications for healthcare resource use. Disclosures P. Spearpoint: Corporate appointments; Employee of Vifor Pharma. C. Sammon: Corporate appointments; Employee of PHMR. A. Ramirez de Arellano Serna: Corporate appointments; Employee of Vifor Pharma. P. Rutherford: Corporate appointments; Employee of Vifor Pharma. Shareholder/stock ownership; Vifor Pharma.

scholarly journals Low-dose versus standard-dose alteplase in acute ischemic stroke in Asian stroke registries: an individual patient data pooling study

2019 ◽  
Vol 14 (7) ◽  
pp. 670-677
Author(s):  
Xia Wang ◽  
Jingwei Li ◽  
Tom J Moullaali ◽  
Keon-Joo Lee ◽  
Beom Joon Kim ◽  
...  
Keyword(s):  
Ischemic Stroke ◽  
Intracerebral Hemorrhage ◽  
Acute Stroke ◽  
Acute Ischemic Stroke ◽  
Real World ◽  
Low Dose ◽  
Standard Dose ◽  
Stroke Registry ◽  
Symptomatic Intracerebral Hemorrhage ◽  
Intravenous Alteplase

Objective To investigate the comparative efficacy and safety of the low-dose versus standard-dose alteplase using real-world acute stroke registry data from Asian countries. Methods Individual participant data were obtained from nine acute stroke registries from China, Japan, Philippines, Singapore, South Korea, and Taiwan between 2005 and 2018. Inverse probability of treatment weight was used to remove baseline imbalances between those receiving low-dose versus standard-dose alteplase. The primary outcome was death or disability defined by modified Rankin Scale scores of 2 to 6 at 90 days. Secondary outcomes were symptomatic intracerebral hemorrhage and death. Generalized linear mixed models with the individual registry as a random intercept were performed to determine associations of treatment with low-dose alteplase and outcomes. Results Of the 6250 patients (mean age 66 years, 36% women) included in these analyses, 1610 (24%) were treated with low-dose intravenous alteplase. Clinical outcomes for low-dose alteplase were not significantly different to those for standard-dose alteplase, adjusted odds ratios for death or disability: 1.00 (0.85–1.19) and symptomatic intracerebral hemorrhage 0.87 (0.63–1.19), except for lower death with borderline significance, 0.77 (0.59–1.01). Conclusions The present analyses of real-world Asian acute stroke registry data suggest that low-dose intravenous alteplase has overall comparable efficacy for functional recovery and greater potential safety in terms of reduced mortality, to standard-dose alteplase for the treatment of acute ischemic stroke.

scholarly journals P129: A phase IV real world study on the use of low dose methoxyflurane (PENTHROX™) for the treatment of moderate to severe trauma pain in the Canadian emergency department (ADVANCE-ED): an interim report on secondary outcomes

CJEM ◽  
2020 ◽  
Vol 22 (S1) ◽  
pp. S111-S111
Author(s):  
S. Campbell ◽  
E. Simard ◽  
A. Arcand ◽  
L. Blagrove ◽  
P. Piraino ◽  
...  
Keyword(s):  
Pain Relief ◽  
Adverse Events ◽  
Acute Pain ◽  
Real World ◽  
Rescue Medication ◽  
Low Dose ◽  
Open Label ◽  
Open Label Study ◽  
Label Study ◽  
Phase Iv

Introduction: Inhaled low dose methoxyflurane (MEOF) was recently approved in Canada for the short-term relief of moderate to severe acute pain associated with trauma or interventional medical procedures in conscious adult patients. ADVANCE-ED is an ongoing phase IV, prospective open label study undertaken to generate real-world evidence to complement the global clinical development program through evaluation of the effectiveness of low dose MEOF in Canadian emergency departments (EDs). Methods: This multi-centre study is enrolling adult (≥18 yrs) patients with moderate to severe acute pain (NRS0-10 ≥ 4) associated with minor trauma. To address limitations from the pivotal study, this study allows patients who were excluded in the pivotal trials: namely, those with severe (≥7) pain, and those using OTC or stably dosed analgesics for other conditions, including chronic pain. Eligible patients receive a single treatment of up to 2 x 3 mL MEOF (2nd 3 mL to be provided only upon request), self-administered by the patient under medical supervision. Rescue medication is permitted at any time, if required. Results: Here we describe the patient demographics and treatment satisfaction (Global Medication Performance, GMP) at 50% enrolment (n = 49). Mean (SD) patient age is 48.0 (17.1) yrs and 55.1% are female. Mean pain (SD) reported at enrolment is 8.3 (1.5), with 73.4% of patients with NRS0-10 ≥ 8. Injuries are overwhelmingly limb trauma (87.8%). The most common type is sprain/strain (40.8%), followed by fracture (32.7%). At 5 minutes post-start of administration (STA) of MEOF, 80.4% of patients reported pain relief; this increased to 91.3% at 15 minutes, and 100% of patients reported pain relief by 30 minutes post-STA. GMP was assessed as “good”, “very good” or “excellent” by ≥80% of patients both 20 minutes post-start of administration (STA) of MEOF (83.3%) and at discharge (85.8%). When asked to what extent their expectation of pain relief had been met, 32.7% responded good, 26.5% responded “very good” and 22.4% responded “excellent”. Three quarters of enrolled patients (75.5%) did not require rescue medication. The most common (≥5%) treatment-related adverse events were dizziness (n = 14, 28.6%) and euphoric mood (n = 4, 8.2%). No serious adverse events have been reported. Conclusion: Based on 50% of the patients enrolled in this prospective, open label study, responses to inhaled low-dose MEOF are within expectation for both effectiveness and tolerability.

scholarly journals Protocolized High-Dose (HD) and Low-Dose (LD) Spinal Cord Stimulation (SCS) Workflow Results in Meaningful Pain and Opiate Reduction

Neurosurgery ◽  
2019 ◽  
Vol 66 (Supplement_1) ◽  
Author(s):  
Sarah E Hodges ◽  
Shervin Rahimpour ◽  
Luis Alejandro Antezana ◽  
Abena A Ansah-Yeboah ◽  
Rajeev Dharmapurikar ◽  
...  
Keyword(s):  
Spinal Cord ◽  
Pain Relief ◽  
Real World ◽  
Spinal Cord Stimulation ◽  
Low Dose ◽  
High Dose ◽  
Pain Medications ◽  
Long Term Follow Up ◽  
Permanent Implant

Abstract INTRODUCTION Various new waveforms for spinal cord stimulation (SCS) have emerged in recent years, with limited data supporting their utility in a real-world clinical setting. We report real-world results of a protocolized workflow algorithm that allows for high dose (HD) and low dose (LD) neurostimulation in patients with chronic pain undergoing SCS trial or permanent procedures. METHODS Prospective data was collected using the ManageMySurgery (MMS) mobile device platform in patients undergoing Medtronic SCS trial and permanent implant procedures. E-consent was obtained through the HIPAA compliant, mobile software platform. All data was de-identified, aggregated and analyzed. RESULTS In total, 104 patients (37 trial SCS and 67 permanent SCS) participated. For SCS trial and permanent procedures, the protocolized workflow algorithm resulted in a 91% trial success rate with >50% pain relief. At long-term follow-up (3 to 12 mo), 86% of permanent SCS patients reported they were getting the same or more relief as during their SCS trial. For permanent SCS patients, 79% reported >50% improvement in overall pain and 58% had >50% improvement in low back pain. The protocolized workflow algorithm resulted in a 37% “remitter rate,” with these patients reporting themselves essentially pain free (VAS 0–3). Importantly, 52% of permanent implant patients stopped or reduced their ‘as needed’ pain medications by >50%. Additionally, 87% would recommend the same procedure to a friend or family member, 87% found device charging ‘easy’ or ‘very easy’ and 66% reported charging a few times a week or weekly. CONCLUSION A protocolized workflow algorithm that allows for HD and LD neurostimulation appears to have robust utility in providing meaningful pain relief and opiate reduction during both the SCS trial and permanent stages and at longer-term follow-up. Randomized controlled trials with extended follow-up are in progress.

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