Immunoglobulin replacement for primary immunodeficiency: Indications for initiating and continuing treatment

Background: Immunoglobulin replacement therapy (IGRT) is the foundation of treatment for the majority of patients with primary immunodeficiency. Clinical history and laboratory evaluation define the patients for whom IGRT is necessary and appropriate. During the 70 years since the first patient was treated, new products have led to the development of several modes of administration that facilitate the individualization of treatment that enables the optimization of care. Objective: The objective was to explain the assessment of candidates for IGRT and approaches to reevaluating recipients of IGRT to decide on the need to continue treatment and to review the approaches to optimize IGRT. Methods: The relevant literature was reviewed in the context of the author's experience supervising > 20,000 IGRT treatments over a 40-year period. Results: Providing the most appropriate form of IGRT for individual patients ameliorates disease and lessens the burden of care for patients with primary immunodeficiency. Conclusion: IGRT is safe and effective when used to treat patients with primary immunodeficiency who meet established and appropriate clinical and laboratory criteria.

Health and functional status of tiotropium/olodaterol-treated patients with COPD: results from the AERIAL® non-interventional study

Patients with chronic obstructive pulmonary disease (COPD) often have reduced physical activity, which can impair health status. Real-world data can provide valuable information on the health and functional status of patients with COPD treated with tiotropium/olodaterol.AERIAL® (NCT03165045) was a German, non-interventional study of patients with COPD receiving treatment with tiotropium/olodaterol under real-world conditions for approximately 6 weeks. The primary endpoint was the proportion of patients achieving a decrease of ≥0.4 points in Clinical COPD Questionnaire (CCQ) score. The CCQ-4 subdomain was used to assess functional status, and the Physician's Global Evaluation (PGE) scale to assess the patients’ general condition. Safety was also assessed, as well as patient satisfaction and willingness to continue treatment.Of 1351 screened patients, 1322 were treated and 1140 comprised the full analysis set. The primary endpoint was met: 66.3% of patients achieved a ≥0.4-point decrease in overall CCQ score (mean decrease±standard deviation [sd] 0.78±0.95). Mean decrease ±sd in CCQ symptoms and functional state subdomains were 0.84±1.06 and 0.75±1.05 points, respectively. PGE scores improved. One fatality (not treatment-related) and 23 drug-related adverse events were recorded, most commonly nausea and vertigo. Over 85% of patients were satisfied/very satisfied with tiotropium/olodaterol overall and with the Respimat device, both in terms of inhalation and handling. Most patients (95.2%) expressed willingness to continue treatment.Patients with COPD treated with tiotropium/olodaterol via Respimat in routine clinical practice had clinically relevant improvements in health and functional status compared with baseline.

Ethical Issues, Discrimination and Social Responsibility Related to HIV-Infected Patients

The aim of the present study was to assess the attitude of students attending 4th and 6th year of the Faculty of Dental Medicine in Iasi towards HIV-seropositive patients. A questionnaire was elaborated, containing 14 ethic statements related to some aspects they may encounter in their future career: the observance of confidentiality and the rights of HIV/AIDS infected patients, the refusal of dedicated treatment and the appreciation of discrimination in this situation. The results have identified some negative aspects regarding the above mentioned issues, mainly in terms of the refusal of specialized treatment. A percentage between 47.4% and 38.4% of the students strongly agreed to the affirmations according to which the risk of cross-infection and the lack of some safety conditions at work may be a reason for the refusal to provide dental treatment. The responses referring to discrimination suggest that there is a negative attitude towards the following aspects: the treatment of HIV-infected patients in different offices, the refusal to continue treatment after confirmation of the diagnosis, the refusal to cooperate with an infected colleague. Only 57.8% of the subjects considered the refusal of treatment as discrimination, with no statistical differences between the years of study. The results suggest both the need to modify the academic curriculum by introducing some ethics and medical legislation courses, and the adoption of a strategy for the increase of empathy and social responsibility in relation to this group of discriminated persons.

Treating patients with a new coronavirus infection in the clinic of Izmerov Research Institute of Occupational Health

Introduction. To comply with the order of the Government of the Russian Federation and the Ministry of Education and Science, the clinic of Izmerov Research Institute of Occupational Health received the necessary conditions for the provision of medical care to patients with a new coronavirus infection. The study aims to identify possible criteria for the positive dynamics of pneumonia caused by the SARS-CoV-2 virus and to describe the general characteristics of patients hospitalized in the COVID center of Izmerov Research Institute of Occupational Health. Materials and methods. The article describes the clinical, laboratory, and instrumental data of 68 patients with pneumonia caused by the SARS-CoV-2 virus hospitalized to continue treatment. Statistical analysis included descriptive statistics. The significance level was less than 0.05. Results. The COVID-center received patients with complaints of shortness of breath on exertion, an unproductive cough, general weakness, myalgias, headaches, and an impaired sense of smell and taste. The condition of the patients was moderate. O2 saturation in ambient air was 93% and higher. Computed tomography of the chest revealed lesions of the lung tissue (numerous peripheral seals of the "frosted glass" type of various shapes and lengths with areas of consolidation), mainly corresponding to bilateral polysegmental viral pneumonia CT 1-2 degrees. The most frequent changes in laboratory parameters in patients were increased values of ESR (51.16% of cases), D-dimer (50% of cases), CRP (22.06% of cases), relative values of monocytes (58.14% of cases), as well as low hemoglobin concentrations (23% of cases). Conclusions. In patients with a new coronavirus infection hospitalized to continue treatment in the COVID center of Izmerov Research Institute of Occupational Health, lung tissue lesions were detected, mainly corresponding to the moderate-severe form of bilateral polysegmental viral pneumonia (CT of the 1-2 degree). The most frequent changes in laboratory parameters in patients with COVID-19 were increased ESR values, D-dimer, CRP, relative values of monocytes, and low hemoglobin concentrations. Monocytes, being one of the indicators of activation of the immune defense, are a laboratory criterion for the positive dynamics of the course of new coronavirus infection.

SPIRITUALITY AND ITS REFLECTIONS IN ONCOLOGICAL PATIENTS

Since the dawn of mankind, science and faith have always been given as antonyms and have fought for popular recognition. However, these days the association between these areas is discussed for a better result, since believing in one of these aspects does not mean disregarding the importance of the other, even in terminal patients. The objective was to analyze the experience of spirituality/religion in supporting cancer patients and to discuss the lack of preparation and use of these tools by health professionals. The literature review was based on the BVS Brazil (Virtual Health Library), Google Scholar and PubMed databases from 2017 to 2020. The keywords used were “spirituality”, “religion”, “neoplasms” and "health". As an inclusion criterion, free full texts in English were used. The scientific search initially showed 86 articles where after the evaluation of their titles and respective abstracts, duplicates and those that were incompatible with the scope of the study were excluded, totaling 31 articles. After attaching an article separately, 31 articles were fully read for the construction of this work. Concluding that spirituality and religion are important in the lives of terminally ill patients who seek tools to cope with the disease and to continue treatment, showing the importance of these in the way of dealing with the patient, thus improving the prognosis.

TO ASSESS THE KNOWLEDGE IN SELF CARE PRACTICE AMONG PATIENT SEEKING HEMODIALYSIS WITH ARTERIVENOUS FISTULA

People with failed or damaged kidneys may have difficulty in eliminating waste and unwanted water from the blood. Dialysis is an artificial way of carrying out this process. Dialysis substitutes the natural work of the kidneys. Hemodialysis is the most common and durable treatment for ESRD patients. However, this changes patient’s life completely by imposing certain constrains on their routine activities affecting the compliance to therapy, so they need assistance to continue treatment. A proper vascular access is the key for effective hemodialysis. The results of the descriptive study is very less number of the patient having knowledge about self-care practices of fistula. After the completion of this study researcher planned for the enhance of the knowledge about the self-care practices of fistula by pamphlet and learning programme etc.

Low-dose Psoralen–UV-A maintenance therapy prolongs disease-free remission rates in mycosis fungoides patients

A clinical decision report using Vieyra-Garcia P, Fink-Puches R, Porkert S, et al. Evaluation of Low-Dose, Low-Frequency Oral Psoralen–UV-A Treatment With or Without Maintenance on Early-Stage Mycosis Fungoides. JAMA Dermatology. 2019;155(5):538. https://doi.org/10.1001/jamadermatol.2018.5905 for a patient weighing whether to continue treatment in light of socioeconomic circumstances.

Analysis of Hospitalization Events in Patients with Primary Hemophagocytic Lymphohistiocytosis (HLH) Treated with Emapalumab

Background Primary HLH is a rare, genetic immune-mediated disorder, usually diagnosed during infancy or early childhood. It is characterized by severe and often life-threatening immune activation, hyperinflammation and immune-mediated organ damage. Like some other rare diseases, the epidemiology of primary HLH is not well defined. Published data suggest the annual incidence of primary HLH is 1.8 per 100,000 live births in Sweden (Meeths et al. Pediatr Blood Cancer 2015). Current conventional treatment for primary HLH includes chemoimmunotherapy to suppress the immune hyperactivation and then offering patients the only potentially curative treatment, namely allogenic hematopoietic stem cell transplantation (HSCT). Chemoimmunotherapy (as recommended in the HLH-94 and HLH-2004 treatment protocols) is based on the combined use of dexamethasone, etoposide and cyclosporine. The side effects associated with these drugs are expected to increase the burden of the illness. Emapalumab, a fully human monoclonal antibody that neutralizes IFN-gamma, is approved in the USA for use in adult and pediatric patients with primary HLH with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Patients with primary HLH are likely to be hospitalized for extended periods during diagnosis and chemoimmunotherapy (8-week induction course followed by continuation phase until HSCT). Most published data for primary HLH focus on the treatment and management of HLH in treatment-naïve patients; data from the largest prospective study on HLH reported that the median time to HSCT was 148 days (range, 25-2105) (Bergsten et al. Blood 2017). We performed a post-hoc analysis of the NI-0501-04/05 study, which followed patients treated with emapalumab for up to 1-year post-HSCT, to assess the patterns of hospitalization. Methods Emapalumab was evaluated in an open-label, single-arm, multicenter phase 2/3 trial in patients aged ≤18 years with suspected or confirmed primary HLH (NCT01818492; Locatelli et al. N Engl J Med 2020). Patients were treatment-naïve or -experienced, with active disease at enrollment; treatment-experienced patients had shown worsened/reactivated disease, poor response to conventional therapy, or were unable to continue treatment due to adverse events. Emapalumab was administered as an intravenous infusion at an initial dose of 1 mg/kg every 3 days and subsequent doses could be increased to 3, 6 and 10 mg/kg if required. Treatment duration was 8 weeks, with possible shortening to a minimum of 4 weeks, or extension up to the time of HSCT if needed. Patients were required to stay in hospital initially for 2 weeks, and then could return home whilst awaiting HSCT at the discretion of the treating physician. Patients were invited to participate in a long-term follow-up study for up to 1 year after the last dose of emapalumab or after HSCT. They could continue treatment with emapalumab beyond week 8 if HSCT was delayed and the benefit-risk ratio was considered favorable (NCT02069899). Post-hoc analyses were conducted using data from all patients who received of ≥1 dose of emapalumab. Descriptive analyses were performed for the hospitalization data collected. Results The study population comprised treatment-naïve (n=7) and treatment-experienced patients (n=27). All patients were hospitalized for at least 2 weeks as per the trial protocol. The mean (standard deviation [SD]) duration of hospitalization until HSCT, death or end of study (EOS) was 83 (47) and 64 (35) days in treatment-naïve and treatment-experienced patients, respectively (Table 1). Twenty (59%) patients were discharged while waiting for HSCT. The mean (SD) period of time spent at home until HSCT, death or EOS was 164 (212) and 61 (106) days in treatment-naïve and treatment-experienced patients, respectively. Conclusions Over 50% of treatment-naïve and -experienced patients with HLH who received emapalumab were permitted to be at home for an average of 12 weeks. Reduced length of hospitalization is expected to lead to improved patient health-related quality of life, reduced burden on patients and caregivers, and reduced healthcare utilization and costs. These factors are intended to be explored within future research studies. Disclosures Wilson: Sobi: Current Employment, Current equity holder in publicly-traded company. Pochopien:Sobi: Consultancy. Nazir:Sobi: Current Employment. Rao:Sobi: Consultancy, Other: Advisory Board. Locatelli:Miltenyi: Speakers Bureau; Bellicum Pharmaceutical: Membership on an entity's Board of Directors or advisory committees; Novartis: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Amgen: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Jazz Pharmaceeutical: Speakers Bureau; Medac: Speakers Bureau.

Evaluation of attitude among infertile couples about continuing assisted reproductive technologies therapy during novel coronavirus outbreak

Study question: Does the fear of the coronavirus disease 2019 (COVID-19) pandemic reduce the desire of infertile couples to continue treatment? Summary answer: Most of the participants in this study wanted to continue treatment. What is known already: The effect of the prevalence of infectious diseases including the Zika virus on the attitude of infertile couples has been studied in very few studies. However, the effect of the outbreak of COVID-19 on the attitude of infertile couples has not been investigated. Study design, size, duration: We conducted a prospective longitudinal study on forty-six infertile couples (n=92) who referred to our infertility clinic from 4 March 2020 through 20 June 2020. Participants/materials, settings, methods: This study is based on potential infertile couples for treatment with assisted reproductive technology (ART) who referred to our infertility clinic, Sari, Iran (median age of 35.5 years). All individuals with primary infertility, as defined by the World Health Organization (WHO) and candidates for ART, were included in the study. People who did not agree to participate in the study were excluded. Subjects were surveyed using a researcher-made questionnaire. This questionnaire has four sections as follows: The first part included demographic information and clinical characteristics, the second part included medical records, the third part included questions related to assessing the level of awareness regarding coronavirus infection, and the fourth part included questions related to the attitude towards continuing infertility treatment. The validity of this questionnaire was assessed by three infertility specialists and was confirmed with Cronbachs alpha of 0.78. A P-value of less than 0.05 was considered statistically significant. Main results and the role of chance: There is no significant relationship between COVID-19 symptoms and the level of awareness (P-value <0.05). Thirty-two patients (33.33%) had decreased motivation to continue treatment during COVID-19 pandemic. Fear of transmission to the fetus (28.13%) had the highest frequency among the causes of decreased motivation to continue treatment (P-value = 0.011). Confidence in support of the treatment team (56.67%) was the most common reason for lack of motivation in people without decreased motivation (P-value <0.001). The majority of patients had a very high or high tendency (65.22%) to continue or start treatment during the COVID-19 pandemic (P-value <0.001). Most people had an average fear of getting the disease (39.13%) (value <0.001). Examining the relationship between the presence of COVID-19 symptoms and attitude level variables showed that there is only a significant relationship between the greater desire to have a child and the continuation or initiation of treatment with the presence of COVID-19 symptoms (P-value = 0.032). Limitations, reasons for caution: We were not able to fully assess patients' deep feelings and cultural beliefs, due to the use of questionnaires and the lack of interviews. Wider implications of the findings: Our results showed for the first time that patients' knowledge about COVID-19 and trust in the treatment staff played an effective role in selecting and continuing infertility treatment. To clarify this issue, studies with the larger statistical community in the form of interviews are needed. Study funding/completing interest(s): The study received financial support from the Mazandaran University of Medical Sciences (Grant number: 7903). None of the funders had any role in the study design, collection, analysis, or interpretation of data, writing of the paper, or publication decisions. The authors have nothing to declare. Trial registration number: N/A