scholarly journals Patient-reported effectiveness and safety of Pamidronate in NSAIDs-refractory chronic recurrent multifocal osteomyelitis in children

2021 ◽  
Author(s):  
Bartłomiej Juszczak ◽  
Jerzy Sułko
Keyword(s):  
Adverse Drug Reactions ◽  
Social Life ◽  
Chronic Recurrent Multifocal Osteomyelitis ◽  
Daily Activities ◽  
Drug Reactions ◽  
Patient Reported ◽  
Multifocal Osteomyelitis ◽  
Drug Dependent ◽  
Treatment Onset ◽  
The Impact

AbstractTo evaluate patient-reported effectiveness, safety and social influence of Pamidronate in the therapy of NSAIDs-refractory Chronic Recurrent Multifocal Osteomyelitis in children. Authors reviewed self-created questionnaires, which asked patients for symptoms alleviation, adverse drug reactions frequency and degree of severity and daily activities self-reliance. Only surveys with complete answers, which were returned to authors by an e-mail from juvenile patients treated for NSAIDs-refractory Chronic Recurrent Multifocal Osteomyelitis at the University Children’s Hospital of Cracow were analyzed. Between 2010 and 2019, 61 children were diagnosed with NSAIDs-refractory Chronic Recurrent Multifocal Osteomyelitis at our department. Out of 61 requests sent, 42 complete replies (33 females, 9 males) were gathered and analyzed. All patients included in this research were administered with at least one set of Pamidronate intravenously in the dose of 1 mg/kg/day for 3 consecutive days. Our analysis shows remarkable in terms of patient’s impressions decrease of pain intensity after 2.5 series of Pamidronate on average, and total pain resolution after 5.9 series on average. Overall number of adverse drug reaction events reported by responders was 105. One patient developed drug-dependent renal insufficiency in the course of therapy. Outcome assessment indicates that nearly 50% of the studied population was more eager to participate in social life just after the first infusion of the drug. 95% of the surveyed unanimously agreed to recommend Pamidronate therapy to cure NSAIDs-refractory CRMO. 39 out of 42 (93%) patients considered Pamidronate effective at the end of the treatment. Onset of Pamidronate’s action is gradual and differs in terms of symptoms alleviation between sexes. The therapy can induce considerable number of adverse drug reactions (2.5 per patient). Only 3 out of 42 (7%) patients were free from any ADRs. To demonstrate the impact of the use of Pamidronate on daily activities more precisely, further research with quantification of the quality of life is warranted.

scholarly journals P330 Inflammatory Bowel Disease (IBD) patients frequently report adverse drug reactions during biologic therapy: A multicentre, prospective, patient-reported pharmacovigilance monitoring system

2020 ◽  
Vol 14 (Supplement_1) ◽  
pp. S319-S319 ◽  
Author(s):  
P THOMAS ◽  
R West ◽  
M Russel ◽  
J Jansen ◽  
J van Lint ◽  
...  
Keyword(s):  
Healthcare Provider ◽  
Monitoring System ◽  
Adverse Drug Reactions ◽  
Daily Practice ◽  
Drug Reactions ◽  
Therapeutic Consequences ◽  
Treatment Optimisation ◽  
Patient Reported ◽  
High Level ◽  
System Organ

Abstract Background The use of biologicals has improved the treatment of IBD but the understanding of adverse drug reactions (ADRs) and the knowledge of patients’ perception on ADRs is poor. Patient-reporting may provide more insight in the extent and burden of ADRs in daily practice which in turn can lead to treatment optimisation. This study aimed to assess systematic patient-reported ADRs during biological therapy in IBD patients. Methods This multicentre, prospective, event monitoring study enrolled adult Crohn’s disease (CD) and ulcerative colitis (UC) patients treated with a biological between 1 January 2017 and 31 December 2018. Patients completed bimonthly comprehensive web-based questionnaires regarding indication and use of, description of biological induced ADRs, follow-up of previous ADRs, experienced burden of the ADR using a 5-point Likert scale, contact with a healthcare provider and therapeutic consequences due to the ADR. Patient-reported ADRs were MedDRA coded by trained pharmacovigilance assessors. MedDRA is a multi-hierarchical dictionary used to code reported ADRs into specific unambiguous terms (preferred terms). Preferred terms are subsequently grouped into high-level terms, high-level group terms and system organ classes. In total there are 26 system organ classes. Results In total, 182 patients in 4 centres (female 51%, mean (standard deviation) age 42.2 (14.2) years, CD 77%) were included and completed 750 questionnaires. At baseline, 49% used an immunomodulator (43% thiopurines, 6% methotrexate), while biologicals were documented as follows: 59% infliximab, 30% adalimumab, 9% vedolizumab, 1% ustekinumab. At least one ADR was reported by 50% of the participants, and 233 ADRs were reported in total with a median reported ADRs per participant of 2 (interquartile range, 1–3). Fatigue (n = 26), headache (n = 20), injection site reactions (n = 16) and arthralgia (n = 12) were most commonly reported, with a mean burden of 3.31, 1.63, 2.55 and 3.33, and a correlation in time with the administration of the biologic was described in 58%, 85%, 81% and 8% in these ADRs, respectively. Participants contacted a healthcare provider in 62% of all ADRs. In two out of 90 patients who reported an ADR, the biological was discontinued. Conclusion We established a patient-reported pharmacovigilance monitoring system and participants in this study frequently reported ADRs due to biologicals. Fatigue and arthralgia resulted in the highest burden. This reporting system may provide more understanding of patient-experienced ADRs which may ultimately lead to increased adherence of therapy and improved quality of life.

scholarly journals Body Weight as a Determining Factor in the Predominance of Adverse Drug Reactions Induced by Fixed-Dose Adalimumab Injections in Female Patients in a Korean Hospital Setting

2020 ◽  
Vol 9 (2) ◽  
pp. 461
Author(s):  
Kwi Suk Kim ◽  
Young Hee Choi ◽  
Aree Moon ◽  
Sang Geon Kim
Keyword(s):  
Body Weight ◽  
Adverse Drug Reactions ◽  
Laboratory Data ◽  
Fixed Dose ◽  
Drug Reactions ◽  
Female Patients ◽  
Adalimumab Therapy ◽  
Male Patients ◽  
The Impact ◽  
Determining Factor

Adalimumab is used at 40-mg dose to treat systemic inflammatory diseases. Given the impact of adverse drug reactions (ADRs), which particularly result in the discontinuation of adalimumab therapy in female patients, this study examined whether sex affects the frequency and type of ADRs induced by adalimumab. In this study, the prescription records and laboratory data of patients aged ≥19 years who had been admitted to the Seoul National University Hospital (SNUH) and prescribed adalimumab were analyzed using an electronic medical record database. The analysis revealed that female patients more frequently experienced adalimumab-induced ADRs compared with male patients (63.2% vs. 52.2%). The incidence of ADRs was significantly higher in female patients with ankylosing spondylitis or rheumatoid arthritis than in male patients with similar conditions (81.5% vs. 60.7% or 64.4% vs. 50.0%, respectively). The median body weight (BW) was lower in female patients than in male patients (54.0 vs. 66.0 kg). Moreover, the incidence of ADRs in patients with a BW of <54.0 kg (i.e., the median female BW) was higher than for those with a BW of ≥54.0 kg, in both males and females. Our results suggested that the predominance of ADRs induced by adalimumab in females was because of their relatively lower BW. This suggests the importance of BW as a determining factor in sex disparity of ADR occurrences.

scholarly journals Assessment of the impact of mitochondrial genotype upon drug-induced mitochondrial dysfunction in platelets derived from healthy volunteers

2021 ◽  
Author(s):  
Amy L. Ball ◽  
Katarzyna M. Bloch ◽  
Lucille Rainbow ◽  
Xuan Liu ◽  
John Kenny ◽  
...  
Keyword(s):  
Mitochondrial Dysfunction ◽  
Healthy Volunteers ◽  
Adverse Drug Reactions ◽  
Mitochondrial Function ◽  
Flux Analysis ◽  
Drug Reactions ◽  
Drug Induced ◽  
Mtdna Variation ◽  
Mitochondrial Genotype ◽  
The Impact

AbstractMitochondrial DNA (mtDNA) is highly polymorphic and encodes 13 proteins which are critical to the production of ATP via oxidative phosphorylation. As mtDNA is maternally inherited and undergoes negligible recombination, acquired mutations have subdivided the human population into several discrete haplogroups. Mitochondrial haplogroup has been found to significantly alter mitochondrial function and impact susceptibility to adverse drug reactions. Despite these findings, there are currently limited models to assess the effect of mtDNA variation upon susceptibility to adverse drug reactions. Platelets offer a potential personalised model of this variation, as their anucleate nature offers a source of mtDNA without interference from the nuclear genome. This study, therefore, aimed to determine the effect of mtDNA variation upon mitochondrial function and drug-induced mitochondrial dysfunction in a platelet model. The mtDNA haplogroup of 383 healthy volunteers was determined using next-generation mtDNA sequencing (Illumina MiSeq). Subsequently, 30 of these volunteers from mitochondrial haplogroups H, J, T and U were recalled to donate fresh, whole blood from which platelets were isolated. Platelet mitochondrial function was tested at basal state and upon treatment with compounds associated with both mitochondrial dysfunction and adverse drug reactions, flutamide, 2-hydroxyflutamide and tolcapone (10–250 μM) using extracellular flux analysis. This study has demonstrated that freshly-isolated platelets are a practical, primary cell model, which is amenable to the study of drug-induced mitochondrial dysfunction. Specifically, platelets from donors of haplogroup J have been found to have increased susceptibility to the inhibition of complex I-driven respiration by 2-hydroxyflutamide. At a time when individual susceptibility to adverse drug reactions is not fully understood, this study provides evidence that inter-individual variation in mitochondrial genotype could be a factor in determining sensitivity to mitochondrial toxicants associated with costly adverse drug reactions.

Improving Ambulation in Multiple Sclerosis

US Neurology ◽  
2009 ◽  
Vol 05 (01) ◽  
pp. 50
Author(s):  
Francois Bethoux ◽  
Keyword(s):  
Multiple Sclerosis ◽  
Walking Speed ◽  
Outcome Measurement ◽  
Daily Activities ◽  
Walking Distance ◽  
Patient Reported ◽  
Walking Performance ◽  
Patient Reported Measures ◽  
The Impact

Ambulation is frequently affected by multiple sclerosis (MS), and is one of the most valued neurological functions among individuals with MS. While walking speed and walking distance have been used for decades as indicators of disease progression, other aspects of gait disturbance are not routinely assessed, and the impact of walking limitations on the daily activities and quality of life of patients is not fully understood. Recently, rehabilitation techniques, devices, and medications that aim directly at improving walking performance have been tested in individuals with MS. At the same time, clinician-rated and patient-reported measures of ambulation are being validated in this patient population. As a consequence of these advances, clinicians can draw from a growing body of evidence to enhance decision-making and outcome measurement when trying to help MS patients fight one of the most visible consequences of their disease.

scholarly journals Physicians’ perspectives on adverse drug reactions in pediatric routine care: a survey

2021 ◽  
Author(s):  
Martina P. Neininger ◽  
Sarah Jeschke ◽  
Lisa M. Kiesel ◽  
Thilo Bertsche ◽  
Astrid Bertsche
Keyword(s):  
Adverse Drug Reactions ◽  
Pediatric Patients ◽  
Routine Care ◽  
Drug Prescribing ◽  
Off Label Use ◽  
Drug Reactions ◽  
Online Questionnaire ◽  
Black Triangle ◽  
Parent Counseling ◽  
The Impact

Abstract Background Children and adolescents are at particular risk for adverse drug reactions (ADRs). We investigated physicians’ perceptions on ADRs in pediatric routine care. Methods In this exploratory study from April to November 2020, we invited physicians treating pediatric patients across Germany to complete an online questionnaire consisting mainly of closed questions. Results Completion rate was 98% (127/129). Of all participants, 23% (29/127) stated they were not able to estimate how many of their pediatric patients experienced ADRs during drug therapy. The remaining physicians estimated that 7.5% (median; Q25/Q75 3%/20%) of their pediatric patients were affected by ADRs. Regarding counseling on ADRs, 61% (77/127) stated they do not ask regularly the extent to which parents want to be informed. In total, 26% (33/127) stated they avoid counseling on ADRs concerning commonly used approved therapies, whereas only 4% (5/127) did so concerning off-label use (P < 0.001). Altogether, 16% (20/127) stated they rather prescribe new medicines as they hope for better effectiveness; 72% (91/127) said they are cautious about doing so owing to yet unknown ADRs. Of all respondents, 46% (58/127) stated they do not report ADRs to the authorities. Concerning the black triangle symbol, a European pharmacovigilance measure, 11% (14/127) stated they knew it and 6% (7/127) stated they reported any suspected ADR for drugs with that symbol. Conclusions Physicians’ perspectives on ADRs were ambivalent: ADRs influenced their parent counseling and drug prescribing; yet, they struggled to estimate the impact of ADRs on their patients and were not aware of specific pharmacovigilance measures.

scholarly journals Development of the impact of weight on daily activities questionnaire: A patient‐reported outcome measure

2020 ◽  
Vol 10 (6) ◽  
Author(s):  
Claire M. Ervin ◽  
Diane Whalley ◽  
Lisa von Huth Smith ◽  
Rebecca Crawford ◽  
Jennifer Dine ◽  
...  
Keyword(s):  
Outcome Measure ◽  
Patient Reported Outcome ◽  
Daily Activities ◽  
Patient Reported Outcome Measure ◽  
Patient Reported ◽  
The Impact

scholarly journals Development of a novel patient-reported measure for acromegaly: the Acro-TSQ

Pituitary ◽  
2019 ◽  
Vol 22 (6) ◽  
pp. 581-593 ◽  
Author(s):  
Maria Fleseriu ◽  
Leon Fogelfeld ◽  
Murray B. Gordon ◽  
Jill Sisco ◽  
Hilary H. Colwell ◽  
...  
Keyword(s):  
Side Effects ◽  
Disease Burden ◽  
Symptom Control ◽  
Somatostatin Analogs ◽  
Well Being ◽  
The United States ◽  
Daily Activities ◽  
Response Options ◽  
Patient Reported ◽  
The Impact

Abstract Purpose Somatostatin analogs (SSAs) represent a mainstay of medical treatment for acromegaly, currently available as either intramuscular or deep subcutaneous injections. Patient-reported outcomes (PROs) are increasingly common as relevant outcomes in studies of acromegaly and its treatment, but there are no validated PRO measures available that focus on the disease burden and the impact of treatment, specifically designed for use in patients with acromegaly. We sought to develop a new and unique PRO measure, the Acromegaly Treatment Satisfaction Questionnaire (Acro-TSQ). Methods Concept elicitation (CE) interviews were conducted with acromegaly patients in the United States receiving SSA injections at a stable dose for ≥ 6 months. A questionnaire was drafted based on these interviews; combined CE and cognitive debriefing (CE/CD) interviews were then conducted to confirm the content, clarity, and relevance of the questionnaire. Results Nineteen subjects completed interviews [n = 9 CE, n = 10 CE/CD; n = 15 Lanreotide Depot/Autogel (Somatuline), n = 4 Octreotide LAR (Sandostatin LAR)]. Most subjects responded positively when asked about the effectiveness of their current treatment; however, breakthrough symptoms, injection site reactions, and side effects were commonly reported and had negative impacts on social and emotional well-being and daily activities. All 10 subjects involved in debriefing interviews found the questionnaire to be relevant, easy to complete, and found the response options to be clear. The resulting 26-item Acro-TSQ covers symptoms and symptom control, gastrointestinal side effects and their impact on daily activities, the emotional impact of treatment, convenience and ease of use, and overall satisfaction. Conclusions The Acro-TSQ is a novel PRO, focused on both disease burden and impact of treatment; it was found to be comprehensive, clear, and relevant for patients with acromegaly receiving injectable SSA treatment.

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