scholarly journals Facilitated subcutaneous immunoglobulin use in pediatric patients with primary or secondary immunodeficiency diseases

Immunotherapy ◽  
2021 ◽  
Author(s):  
Ulrich Baumann ◽  
Maria Fasshauer ◽  
Christine Pausch ◽  
Helmut Wittkowski ◽  
Corinna Hermann ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Pediatric Patients ◽  
Retrospective Chart Review ◽  
Subcutaneous Immunoglobulin ◽  
Secondary Immunodeficiency ◽  
Real World Data ◽  
Clinical Trial Registration ◽  
Immunodeficiency Diseases ◽  
Review Study ◽  
At Home

Aim: While facilitated subcutaneous immunoglobulin (fSCIG) has been evaluated in pediatric patients with primary immunodeficiency diseases in clinical trials, real-world data are lacking. Materials & methods: This multicenter, retrospective, chart review study assessed fSCIG utilization in 30 patients less than 18 years old, with primary or secondary immunodeficiency diseases. Medical records were reviewed at fSCIG initiation and at 6 months. Results: Most (90%) patients received their first fSCIG infusion at a medical facility; by 6 months, all fSCIG infusions were administered at home by the patient/caregiver, the majority infusing every 3–4 weeks into a single site. No serious adverse drug reactions occurred. Conclusion: This study supports the feasibility and tolerability of administering fSCIG at home to pediatric patients with immunodeficiencies. Clinical Trial Registration: DRKS00015436 ( German Clinical Trials Register )

scholarly journals Safety evaluation of intravenous immunoglobulin in pediatric patients: a retrospective, 1-year observational study

2019 ◽  
Vol 10 ◽  
pp. 204209861987673
Author(s):  
Reem Elajez ◽  
Asmaa Ezzeldin ◽  
Hossamaldein Gaber
Keyword(s):  
Intravenous Immunoglobulin ◽  
Safety Profile ◽  
Chart Review ◽  
Pediatric Patients ◽  
Safety Evaluation ◽  
Retrospective Chart Review ◽  
Risk Category ◽  
Good Safety Profile ◽  
Review Study ◽  
Infusion Protocol

Background: Intravenous immunoglobulin (IVIG) is a pooled human plasma protein that has shown efficacy in treating a variety of disorders. IVIG is generally well tolerated and has a good safety profile. There are various IVIG products available on the market, which results in differences in efficacy and safety profile. The aim of this study was to assess the safety profile of IVIG use in pediatric patients and its association with other predicted factors. Methods: Retrospective chart review study of all pediatric patients who received IVIG as an inpatient at Hamad General Hospital in Qatar during 2014. The occurrence of adverse drug reactions (ADR) was tested for any association with other predicted factors, such as patient age, IVIG dose, brand, and adherence to infusion protocol. Results: A total of 345 IVIG prescriptions were received by pediatric patients during the study period. Most common documented side effects were: fever (5.8%), chills (2.6%), and headache (2%). Renal insufficiency was observed only in six cases, with five of those in ‘Risk’ category according to RIFLE criteria. A hypersensitivity reaction was documented in seven patients, despite being premedicated with paracetamol and/or diphenhydramine and following the infusion protocol. None of the predicted factors were found to be significantly associated with ADR incidence except IVIG brand. Conclusions: IVIG generally has a good safety profile in pediatric patients, with low risk of severe ADR. More studies are needed to evaluate the correlation between ADR and IVIG formulation, taking into account other factors that may affect results.

scholarly journals Real-World Patient Characteristics and Treatment Patterns of Naldemedine for the Treatment of Opioid-Induced Constipation in Patients with Cancer: A Multicenter Retrospective Chart Review Study

Medicina ◽  
2021 ◽  
Vol 57 (11) ◽  
pp. 1233
Author(s):  
Eriko Hiruta ◽  
Yukiyoshi Fujita ◽  
Hisao Imai ◽  
Takashi Masuno ◽  
Shigeki Yamazaki ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Clinical Practice ◽  
Adverse Events ◽  
Real World ◽  
Chart Review ◽  
Retrospective Chart Review ◽  
Treatment Patterns ◽  
Poor Performance Status ◽  
Patients With Cancer ◽  
Review Study

Background and Objectives: Naldemedine is a peripherally acting μ-opioid receptor antagonist that improves opioid-induced constipation. Although clinical trials have excluded patients with poor performance status (PS) and those started on naldemedine early after opioid initiation, clinical practice has used naldemedine for the same patients. Therefore, we investigated the treatment patterns of naldemedine in a real-world setting. Materials and Methods: This was a multicenter, retrospective chart review study of opioid-treated patients with cancer receiving naldemedine. Adverse events that occurred within 7 days of naldemedine initiation were evaluated in those who received one or more doses of the same. Effectiveness was assessed in patients who used naldemedine for more than 7 days. Results: A total of 296 patients satisfied the eligibility criteria, among whom 129 (43.6%) had a PS of ≥3 and 176 (59.5%) started naldemedine within 2 weeks of opioid initiation. Moreover, 203 (79.6%) patients had ≥3 bowel movements per week. Incidences of all grades of diarrhea and abdominal pain were 87 (29.4%) and 12 (4.1%), respectively. No patient had grade 4 or higher adverse events. Conclusions: Although nearly half of the patients receiving naldemedine in clinical practice belonged to populations that were not included in the clinical trials, our results suggested that naldemedine in clinical practice had the same efficacy and safety as that in clinical trials.

scholarly journals Overview of subcutaneous immunoglobulin 16.5% in primary and secondary immunodeficiency diseases

Immunotherapy ◽  
2022 ◽  
Author(s):  
Roger H Kobayashi ◽  
Jiří Litzman ◽  
Syed Rizvi ◽  
Huub Kreuwel ◽  
Sonja Hoeller ◽  
...  
Keyword(s):  
Primary Immunodeficiency ◽  
Primary Immunodeficiency Disease ◽  
Patient Needs ◽  
Subcutaneous Immunoglobulin ◽  
Primary Immunodeficiency Diseases ◽  
Secondary Immunodeficiency ◽  
Specific Patient ◽  
Immunodeficiency Diseases ◽  
Key Characteristics ◽  
Immunodeficiency Disease

Most primary immunodeficiency diseases, and select secondary immunodeficiency diseases, are treated with immunoglobulin (IG) therapy, administered intravenously or subcutaneously (SCIG). The first instance of IG replacement for primary immunodeficiency disease was a 16.5% formulation administered subcutaneously in 1952. While most SCIG products are now a 10 or 20% concentration, this review will focus on SCIG 16.5% products with a historical overview of development, including the early pioneers who initiated and refined IG replacement therapy, as well as key characteristics, manufacturing and clinical studies. In determining an appropriate IG regimen, one must consider specific patient needs, characteristics and preferences. There are advantages to SCIG, such as stable serum immunoglobulin G levels, high tolerability and the flexibility of self-administered home treatment.

ACE2/Angiotensin-(1-7)/Mas Receptor Axis in Human Cancer: Potential Role for Pediatric Tumors

2020 ◽  
Vol 21 (9) ◽  
pp. 892-901 ◽  
Author(s):  
Ana Luiza Ataide Carneiro de Paula Gonzaga ◽  
Vitória Andrade Palmeira ◽  
Thomas Felipe Silva Ribeiro ◽  
Larissa Braga Costa ◽  
Karla Emília de Sá Rodrigues ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Angiotensin Ii ◽  
Pediatric Cancer ◽  
Pediatric Patients ◽  
Human Cancer ◽  
Experimental Studies ◽  
At1 Receptor ◽  
Pediatric Tumors ◽  
Mas Receptor

Background: Pediatric tumors remain the highest cause of death in developed countries. Research on novel therapeutic strategies with lesser side effects is of utmost importance. In this scenario, the role of Renin-Angiotensin System (RAS) axes, the classical one formed by angiotensinconverting enzyme (ACE), Angiotensin II and AT1 receptor and the alternative axis composed by ACE2, Angiotensin-(1-7) and Mas receptor, have been investigated in cancer. Objective: This review aimed to summarize the pathophysiological role of RAS in cancer, evidence for anti-tumor effects of ACE2/Angiotensin-(1-7)/Mas receptor axis and future therapeutic perspectives for pediatric cancer. Methods: Pubmed, Scopus and Scielo were searched in regard to RAS molecules in human cancer and pediatric patients. The search terms were “RAS”, “ACE”, “Angiotensin-(1-7)”, “ACE2”, “Angiotensin II”, “AT1 receptor”, “Mas receptor”, “Pediatric”, “Cancer”. Results: Experimental studies have shown that Angiotensin-(1-7) inhibits the growth of tumor cells and reduces local inflammation and angiogenesis in several types of cancer. Clinical trials with Angiotensin-( 1-7) or TXA127, a pharmaceutical grade formulation of the naturally occurring peptide, have reported promising findings, but not enough to recommend medical use in human cancer. In regard to pediatric cancer, only three articles that marginally investigated RAS components were found and none of them evaluated molecules of the alternative RAS axis. Conclusion: Despite the potential applicability of Angiotensin-(1-7) in pediatric tumors, the role of this molecule was never tested. Further clinical trials are necessary, also including pediatric patients, to confirm safety and efficiency and to define therapeutic targets.

scholarly journals Improving pragmatic clinical trial design using real-world data

2019 ◽  
Vol 16 (3) ◽  
pp. 273-282 ◽  
Author(s):  
Susan M Shortreed ◽  
Carolyn M Rutter ◽  
Andrea J Cook ◽  
Gregory E Simon
Keyword(s):  
Clinical Trials ◽  
Trial Design ◽  
Suicide Attempts ◽  
Real World Data ◽  
Electronic Health Record Data ◽  
Pragmatic Clinical Trial ◽  
Pragmatic Clinical Trials ◽  
Sample Size Calculations ◽  
Record Data ◽  
Electronic Health

Background Pragmatic clinical trials often use automated data sources such as electronic health records, claims, or registries to identify eligible individuals and collect outcome information. A specific advantage that this automated data collection often yields is having data on potential participants when design decisions are being made. We outline how this data can be used to inform trial design. Methods Our work is motivated by a pragmatic clinical trial evaluating the impact of suicide-prevention outreach interventions on fatal and non-fatal suicide attempts in the 18 months after randomization. We illustrate our recommended approaches for designing pragmatic clinical trials using historical data from the health systems participating in this study. Specifically, we illustrate how electronic health record data can be used to inform the selection of trial eligibility requirements, to estimate the distribution of participant characteristics over the course of the trial, and to conduct power and sample size calculations. Results Data from 122,873 people with patient health questionnaire (PHQ) responses, recorded in their electronic health records between 1 July 2010 and 31 March 2012, were used to show that the suicide attempt rate in the 18 months following completion of the questionnaire varies by response to item nine of the PHQ. We estimated that the proportion of individuals with a prior recorded elevated PHQ (i.e. history of suicidal ideation) would decrease from approximately 50% at the beginning of a trial to about 5%, 50 weeks later. Using electronic health record data, we conducted simulations to estimate the power to detect a 25% reduction in suicide attempts. Simulation-based power calculations estimated that randomizing 8000 participants per randomization arm would allow 90% power to detect a 25% reduction in the suicide attempt rate in the intervention arm compared to usual care at an alpha rate of 0.05. Conclusions Historical data can be used to inform the design of pragmatic clinical trials, a strength of trials that use automated data collection for randomizing participants and assessing outcomes. In particular, realistic sample size calculations can be conducted using real-world data from the health systems in which the trial will be conducted. Data-informed trial design should yield more realistic estimates of statistical power and maximize efficiency of trial recruitment.

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