Current treatment options with immunoglobulin G for the individualization of care in patients with primary immunodeficiency disease.

Primary antibody deficiencies require lifelong replacement therapy with immunoglobulin (Ig)G to reduce the incidence and severity of infections. Both subcutaneous and intravenous routes of administering IgG can be effective and well tolerated. Treatment regimens can be individualized to provide optimal medical and quality-of-life outcomes in infants, children, adults and elderly people. Frequency, dose, route of administration, home or infusion-centre administration, and the use of self- or health-professional-administered infusion can be tailored to suit individual patient needs and circumstances. Patient education is needed to understand the disease and the importance of continuous therapy. Both the subcutaneous and intravenous routes have advantages and disadvantages, which should be considered in selecting each patient's treatment regimen. The subcutaneous route is attractive to many patients because of a reduced incidence of systemic adverse events, flexibility in scheduling and its comparative ease of administration, at home or in a clinic. Self-infusion regimens, however, require independence and self-reliance, good compliance on the part of the patient/parent and the confidence of the physician and the nurse. Intravenous administration in a clinic setting may be more appropriate in patients with reduced manual dexterity, reluctance to self-administer or a lack of self-reliance, and intravenous administration at home for those with good venous access who prefer less frequent treatments. Both therapy approaches have been demonstrated to provide protection from infections and improve health-related quality of life. Data supporting current options in IgG replacement are presented, and considerations in choosing between the two routes of therapy are discussed.

Health-related quality of life (HRQL) in immunodeficient adults with selective IgA deficiency compared with age- and gender-matched controls and identification of risk factors for poor HRQL.

PURPOSE: Selective IgA deficiency (SIgAD) is the most common primary immunodeficiency with a prevalence of 1/600 in the general population. Any targeted health-related quality of life (HRQL) study of adults with SIgAD has never been presented. The objectives of the study were to compare HRQL between SIgAD adults and randomly selected age- and gender-matched population controls, and to identify risk factors for poor HRQL. METHODS: Thirty-two SIgAD individuals and 63 controls answered three questionnaires (clinical data, Short Form-36 Health Survey (SF-36), infection-related HRQL) at baseline before undergoing medical/dental examinations and laboratory assessments. HRQL in SIgAD was re-evaluated after 6 and 12 months. RESULTS: Baseline: Selective IgA deficiency individuals reported significantly increased fear of contracting infections (p < 0.01). Those scoring high on fear also perceived significantly poorer physical health (p < 0.01). SF-36 results indicated that SIgAD individuals perceived poorer HRQL, although this was not statistically significant. FOLLOW-UP: Compared with SF-36 responses at baseline, SIgAD individuals reported significantly more pain (p < 0.01) at 6 months, poorer general health (p < 0.05) and summarised physical HRQL (p < 0.01) at 6 and 12 months and decreased vitality at 12 months. The summarised mental scale remained stable over time. Risk factors for poor HRQL: The number of antibiotic treatments during the previous year (p < 0.001), number of daily medications (p < 0.01), allergic rhinoconjunctivitis (p < 0.05), chronic musculoskeletal symptoms at least every week (p < 0.05) and anxiety and/or insomnia (p < 0.05) were identified as independent risk factors for poor HRQL. CONCLUSION: The study highlights the importance of identifying and thoroughly evaluating, educating and following up individuals with SIgAD, as their HRQL may be negatively affected due to health problems possible to prevent and treat.

Clinical symptoms in adults with selective IgA deficiency: a case-control study.

Selective IgA deficiency (SIgAD) is the most common primary immunodeficiency in Caucasians with a prevalence of 1/600 and is generally considered a mild disorder. In this study, the clinical status of 32 adults with SIgAD was investigated and compared to 63 age- and gender matched controls, randomly selected from a population database. The SIgAD individuals reported significantly more often contracting various upper and lower respiratory infections, with 8 (25.0 %) having been diagnosed with ≥1 pneumonia in the preceding two years, compared to one (1.6 %) control (p < 0.001). Furthermore, the SIgAD individuals were found to have increased proneness to infections and increased prevalence of allergic diseases and autoimmunity, with a total of 84.4 % being affected by any of these diseases, compared to 47.6 % of the controls (p < 0.01). This study challenges the common statement of SIgAD being a mild form of immunodeficiency. It also highlights the importance of using matched controls in PID clinical research to better detect clinically important manifestations.

Education for dialysis patients lowers long-term phosphate levels and maintains health-related quality of life.

AIMS: This study aims at biological, knowledgeable, behavioral and health-related quality of life effects of self-dosing of phosphate binders before and after an education program in patients with CKD. MATERIAL AND METHODS: The following 12-month study included 43 patients with CKD Stage 4 - 5 and plasma phosphate levels repeatedly above recommended maximum level. Patients underwent a structured educational program regarding calcium and phosphate balance, food intake and phosphate binders comprising a 60-min session, 3 - 5 times during a 2-month period (intervention). Patients' knowledge on the topics included in the program was assessed before and after the program as well as phosphate levels. RESULTS: The mean plasma phosphate level had decreased significantly directly after the intervention (1.87 mmol/l, p < 0.05) and remained low over time; 1.78 mmol/l at 12 months follow-up (p < 0.001). The patients' self-reported knowledge regarding food intake, calcium/phosphate balance and self-dosing of phosphate binders increased significantly after instruction (p < 0.001). CONCLUSION: A structured education program focusing on calcium and phosphate balance and self-dosing of phosphate binders significantly increased the patients' self-reported knowledge regarding these issues which in turn led to changes in behaviors reflected in decreased long-term plasma phosphate levels.

Rapid subcutaneous immunoglobulin administration every second week results in high and stable serum immunoglobulin G levels in patients with primary antibody deficiencies.

Subcutaneous immunoglobulin G (SCIG) infusions as life-long replacement therapy in patients with primary antibody deficiences (PAD) is being applied increasingly. However, only a few published pharmacokinetic studies are available for this route of administration. Therefore, the pharmacokinetics of a 16% immunoglobulin G (IgG) preparation intended for subcutaneous use were investigated in patients with common variable immunodeficiency and X-linked agammaglobulinaemia. SCIG infusions (200 mg/kg body weight) were administered to 12 adult patients every 14 days for 24 weeks (total of 144 infusions). Pharmacokinetic parameters were determined based on serum IgG trough levels and antibody levels against tetanus. The median half-life of the total serum IgG and for the tetanus antibodies was 40.6 and 23.3 days respectively. Median in vivo recovery of serum IgG and tetanus immunoglobulins were 36% and 46% respectively. Median, preinfusion serum IgG trough levels per patient were high without major variations between infusions and ranged from 7.24 to 7.86 g/l. Safety, in terms of adverse events including systemic adverse reactions and local tissue reactions at infusions sites, was monitored throughout the study. Six mild, local tissue reactions were observed during the study in one patient. No systemic adverse reactions related to the study drug were observed and no serious other adverse event occurred during the study. It is concluded that the bi-weekly SCIG therapy was well tolerated in the study and that it results in high and stable serum IgG levels, offering an alternative therapy regimen to patients suffering from PAD.

The impact of food hypersensitivity reported in 9-year-old children by their parents on health-related quality of life.

BACKGROUND: There are only a few studies on the impact of food hypersensitivity (FHS) in children on health-related quality of life (HRQL). The present study was designed to examine this impact in a population-based birth cohort (BAMSE). METHODS: A nested case-control study was performed within the cohort. The parents of 1378 nine-year-old children filled out a generic questionnaire with 13 subscales (Child Health Questionnaire Parental Form 28 - CHQ-PF28) supplemented with disease-specific questions concerning FHS. There were 212 children with report of FHS. Another 221 children with allergic diseases but not FHS were examined for comparison. Furthermore, the impact of pronounced symptoms of FHS and of increasing levels of food-specific IgE antibodies on HRQL was also analysed. RESULTS: The children with FHS exhibited significantly lower scores on the subscales physical functioning, role/social limitations - physical and general health in the generic instrument. Furthermore, children with food-related symptoms from the lower airways were scored lower on Self Esteem, Parental Impact - time and Family Cohesion. Sensitization per se did not alter these patterns, but high levels of food-specific IgE-antibodies affected mental health and general health negatively. A physician's diagnosis of food allergy did not affect any of the subscales negatively. CONCLUSIONS: Parents reported that FHS exerts a negative impact on the HRQL of 9-year-old children, in particular in children with symptoms from the lower airways or if the FHS is associated with high levels of food-specific IgE-antibodies. Healthcare-givers must put major effort into improving and maintaining the HRQL of these children.

Prognostic factors for health-related quality of life in adults and children with primary antibody deficiencies receiving SCIG home therapy.

The aims of the present study were to evaluate the health-related quality of life (HRQL) and treatment satisfaction (TS) of adults and children with primary antibody deficiencies (PAD) before and after the introduction of subcutaneous immunoglobulin G (SCIG) self-infusions at home and to identify prognostic factors (demographic/social, medical, patient/parent reported) for HRQL. 85 adults and 21 parents of children with PAD answered the SF-36 (adults), CHQ-PF50 (parents), and the LQI (adults and parents) at baseline and following 10 months of weekly self-administered SCIG infusions at home. The SCIG home therapy was associated with significant improvements in HRQL and TS, particularly in patients who had previously received IVIG therapy in hospital settings. Background factors that were found to be associated with HRQL changes in adults were age, serum IgG levels at month 10, concomitant joint/muscle/skeletal disorders, clinical study location and smoking status.

Rapid subcutaneous IgG replacement therapy in children and adults–20 years o clinical experience

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Rapid subcutaneous IgG replacement therapy in children and adults –20 years o clinical experience

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Measuring treatment satisfaction in patients with primary immunodeficiency diseases receiving lifelong immunoglobulin replacement therapy.

BACKGROUND: Treatment satisfaction of patients with primary immunodeficiency diseases receiving hospital-based intravenous (IVIG) or home-based subcutaneous (SCIG) immunoglobulin infusions requires investigation. OBJECTIVE: Evaluation of the properties and suitability of the Life Quality Index (LQI), as an instrument to assess treatment satisfaction. METHODS: Patients received weekly SCIG and completed the LQI, two global treatment satisfaction questions and the CHQ-PF50 (children) or the SF-36 (adults) at baseline and 10 months. The LQI was psychometrically evaluated. RESULTS: The LQI comprised four scales: treatment interference (I), therapy related problems (II), therapy setting (III), treatment costs (IV). Convergent/discriminant validity for scales I, II, III was acceptable, for scale IV moderate. CHQ-PF50 scales behavior, bodily pain, global behavior, global health, mental health, parental impact-emotion significantly correlated with LQI scale II, the family activity scale with LQI scales I, III. SF-36 scale bodily pain significantly correlated with scale III. Internal consistency was good for scales I, II, III, but poor for scale IV. Score values significantly increased for scales I, III, IV in patients switching from IVIG to SCIG. CONCLUSIONS: Three valid LQI scales were determined. Cost-related questions should be removed due to low reliability. Patients-perceived therapy effectiveness and patient-physician/nurse interaction should be included in the instrument.

Symptom distress, functional status and health-related quality of life before high-dose chemotherapy with stem-cell transplantation.

The aims of this study were to describe how a group of patients with different malignant diseases perceived symptom distress (SD), functional status (FS) and health-related quality of life (HRQOL) on admission to the hospital for stem-cell transplantation (SCT), to compare the obtained data regarding FS and HRQOL with similar data from two general-population groups, and to relate the results to disease- and treatment-specific data. Fifty-one patients participated in the study. Three instruments were used to collect data: SFID-SCT, SIP and SWED-QUAL. The majority of the patients (92%) reported ongoing symptoms even before the SCT with tiredness (67%) and anxiety (53%) as the two most commonly reported symptoms. Although tiredness and anxiety were reported to be the most frequently occurring symptoms, these symptoms were not considered to cause that much distress. Instead, vomiting, reduced mobility and fever, although less commonly occurring, were reported as highly distressing when present. Compared with the general-population groups, the patients reported significantly poorer FS and HRQOL but no statistically significant correlations were found between SD, FS or HRQOL and the time since the last chemotherapy cycle or cycles respectively. Patients with advanced disease and patients with multiple myeloma were found to report more SD and poorer FS and HRQOL.

Express subcutaneous IgG infusions: decreased time of delivery with maintained safety.

The aims of the study were to evaluate the safety and feasibility of weekly express subcutaneous replacement IgG self-infusions (E-SCIG, 35 mL/h/syringe driver) in 50 patients and to evaluate their perceptions of the therapy. A total of 4900 E-SCIG infusions at separate infusion sites were given on 1228 treatment occasions. The most commonly reported local tissue reactions were swelling (n = 37), redness (n = 25), and soreness (n = 24). A majority of these patients reported the local reactions as less intense or unchanged compared to those arising from rapid SCIG (20 mL/h) infusions. The patients reported a median score of 16 (visual analogue scale, VAS; 1, not troublesome at all) regarding their overall perceptions of the local reactions. They were positive toward the home therapy regime (median VAS score 96; 100, very positive) and anxious to continue with the E-SCIG infusions (median VAS score 98; 100, very anxious). The E-SCIG method seems to be safe, with few pronounced local reactions, and is appreciated by the patients. Express delivery could also potentially facilitate IgG delivery in a wide variety of diseases, such as autoimmune and autoimmune-like conditions of a neurological or rheumatological character.

Rapid subcutaneous IgG replacement therapy at home for pregnant immunodeficient women.

The use of slow or rapid, subcutaneous, IgG (SCIG) infusions in pregnant women with primary antibody deficiencies has been described earlier, though only in single-case reports. In this study, we have evaluated the effect of rapid, SCIG infusions during 11 pregnancies in nine women [six women with common variable immunodeficiency (CVID), two with IgG-subclass deficiencies, and one with combined IgA and IgG2 deficiency]. The weekly infusions were given by the women themselves at a dose of 100 mg/kg/week throughout the pregnancy. No adverse systemic reactions or pronounced, local tissue reactions were recorded during or after the more than 400 infusions. The 11 babies were healthy and were born after 38-42 weeks of uneventful gestation. The concentrations of maternal S-IgG at the time of delivery in the four women with CVID ranged from 6.0 to 8.3 g/L, with cord/maternal S-IgG ratios of 1.0-1.5. The IgG subclasses were transferred to the infants. The SCIG home-therapy regime was greatly appreciated by the women.

Health-related quality of life, symptom distress and sense of coherence in adult survivors of allogeneic stem-cell transplantation.

This is the first Swedish study to evaluate the health-related quality of life and sense of coherence in adult survivors of allogeneic, haematopoietic stem cell transplantation (HSCT). Twenty-five recipients completed three questionnaires 2-4 years after the transplantation. The questionnaires used were the Sickness Impact Profile (SIP), the Symptom Frequency Intensity and Distress (SFID-BMT) scale and the Sense of Coherence (SOC) scale measuring subjective functional status, symptom distress and coping ability. Impairments in functional status were found, as compared with a population norm. The most common impairments were found in the areas of social interaction and sleep and rest. Eye problems, dry mouth, cough, sexual problems, tiredness, anxiety and changes of taste were symptoms reported by more than half of the patients. Despite impaired functioning and a high incidence of symptoms, the general health was described as quite good or excellent by 80% (n = 20) of the patients. The majority (20/22) had also been able to return to work or to attend school. No difference in the sense of coherence was seen, as compared with the population norm. Functional impairments were significantly correlated to a lower degree of sense of coherence.

Interferon-beta treatment for patients with multiple sclerosis: the patients' perceptions of the side-effects.

The aims of this study were to investigate (i) the self-reported frequency and intensity of systemic side-effects and their impact on the daily lives of patients suffering from Multiple Sclerosis (MS) and undergoing interferon-beta therapy and (ii) the self-reported frequency and perceptions of any local-tissue reactions. Forty patients aged 22 - 59 years (27 females) with relapsing/remitting MS were consecutively recruited for the study (17 on interferon-beta-1a and 23 on interferon-beta-1b). Two self-administered questionnaires were used before and after 1, 4, 8 and 16 weeks of therapy. The interferon therapy was found to be associated with flu-like symptoms. Most systemic side-effects were reported to be mild and to have little impact on the patients' daily lives. Asthenia and fatigue were more often rated as moderate or severe. The most frequently reported local-tissue side-effects were redness and local pain at the injection sites. A considerable inter-individual variation was found among patients regarding the perceptions of both the systemic and local side-effects. This suggests that it is of importance to identify early those patients who may need more support or other interventions to maintain a successful compliance. Multiple Sclerosis (2000) 6 349 - 354

The health-related quality of life of patients suffering from the late effects of polio (post-polio).

In Sweden alone, there are today approximately 10 000-16 500 polio survivors. Between 60% and 80% experience new symptoms several years after the initial attack of poliomyelitis. The aims of this study were to investigate and describe the self-rated health-related quality of life and functional status of a group of Swedish patients with post-polio, to investigate whether any differences within the group could be related to demographic or disease-specific data and to compare the post-polio patients with individuals sampled from the general population. Data were obtained by using two questionnaires, the Swedish Health-Related Quality of Life Questionnaire (SWED-QUAL) and the Sickness Impact Profile (SIP). A total of 150 patients, 86 women and 64 men with median age 61 (20-82) years, were consecutively included. The study showed that the patients mainly reported that their physical, functional status was affected by their post-polio condition. Factors found to be associated with the physical, functional status were age and the number of parts of the body affected by the polio. On comparing the post-polio patients with two samples from the Swedish general population, it was found that the patients reported a poorer functional status and health-related quality of life. The women with post-polio reported more pain, as compared with both the men with post-polio and the women in the general population sample. The family life of the patients - in contrast to their physical abilities - did not seem to be affected by the new deteriorating condition. It is concluded that, owing to the wide range of symptoms, the patients with post-polio need care and support from multidisciplinary teams, including nurses and occupational therapists.

Patients' satisfaction with the care given by district nurses at home and at primary health care centres.

The main purpose of this study was to investigate patients' satisfaction with the care given by the district nurses at home and at the primary health care centres in one area of Greater Stockholm. The questionnaire entitled 'Quality of Care from the Patient's Perspective' (QPP) was used. A total of 168 adult, home-care (HC) and 264 outpatient clinic (OC) patients answered this questionnaire. Maximum median scores were reported for most items designed to measure the four following dimensions: 'medical-technical' competence of the district nurses; 'physical-technical' conditions of the care organization; 'identity orientation' in the attitudes and actions of the district nurses and the 'socio-cultural' atmosphere of the care organization; and the perceived accessibility of nursing care. Some areas were identified as being in need of improvement, for example, pain alleviation, safety of the patients' home environment, the possibility of the patients participating in the decision-making process, the feeling that the care is not based on the patients' desires and needs, and the possibility of always meeting the same district nurse. Differences between HC and OC patients and between sub-groups were found regarding demographic characteristics and self-rated, physical health and psychological well-being. HC and OC patients with poor, self-rated, physical health were identified as being likely to be dissatisfied with the care.

Evaluation of a nurse-run asthma school.

Important aims of the study were to investigate whether an educational program (the 'Asthma School') directed by a nurse led to improved knowledge of the disease, to improved self-medication and self-management and to improved, self-rated, functional status. A total of 32 patients (6 males, 26 females, mean age 43 years) was included. The following methods were used to collect the data before and one year after the Asthma School was completed; two study-specific questionnaires for collecting demographic data and measuring different aspects of the patients' knowledge of the disease and its treatment, monthly diary cards, lung-function tests (FEV1) and the Sickness Impact Profile (SIP) questionnaire. The main results of the study were an improved knowledge of the disease and its treatment, better self-management, i.e. more frequent use of the peak expiratory flow meter (PEF-meter) and use of inhaled bronchodilators on an as-required basis, fewer patients on sick-leave and a better, self-rated, physical health status. However, in spite of these encouraging results, the lungfunction was found to be unaffected, no pronounced changes in the use of asthma drugs could be found and the patients' need for medical care remained the same.