Silvia Riva

Health-related quality of life in patients with haemophilia and inhibitors on prophylaxis with anti-inhibitor complex concentrate: results from the Pro-FEIBA study

Patients with haemophilia A and inhibitors are at high risk for severe bleeding, progression of joint disease and deterioration of health‐related quality of life (HRQoL). To determine the impact of prophylaxis with an activated prothrombin complex concentrate (aPCC) on HRQoL, HRQoL was assessed using the Short‐Form (SF)‐36 Health Survey and the EQ‐5D questionnaire in subjects ≥14 years participating in a prospective, randomized, crossover study comparing 6 months of aPCC prophylaxis with 6 months of on‐demand therapy. Eighteen of 19 patients completed the survey or questionnaire before and after the on‐demand therapy and prophylaxis periods. A general trend towards improved HRQoL after prophylaxis was observed for the 18 evaluable patients in all SF‐36 dimensions except for vitality/energy and physical functioning. After prophylaxis, ‘good responders,’ defined as patients experiencing ≥50% reduction in bleeding, exhibited statistically and clinically significant differences in the physical component score (P = 0.021), role – physical (P = 0.042), bodily pain (P = 0.015), and social functioning (P = 0.036). Similarly, the EQ‐5D health profile showed a trend towards improvement after prophylaxis in all evaluable patients. Among the good responders, improvements did not differ from those observed after on‐demand treatment. EQ visual analogue scale values were slightly improved following prophylaxis for all evaluable patients and the EQ‐5D utility index improved in the good responders only. During prophylaxis, patients missed significantly fewer days from school or work because of bleeding than during on‐demand treatment (P = 0.01). In conclusion, by significantly reducing bleeding frequency in good responders, aPCC prophylaxis improved HRQoL compared with on‐demand treatment.

Interactive sections of an Internet-based intervention increase empowerment of chronic back pain patients: randomized controlled trial.

Background Chronic back pain (CBP) represents a significant public health problem. As one of the most common causes of disability and sick leave, there is a need to develop cost-effective ways, such as Internet-based interventions, to help empower patients to manage their disease. Research has provided evidence for the effectiveness of Internet-based interventions in many fields, but it has paid little attention to the reasons why they are effective. Objective This study aims to assess the impact of interactive sections of an Internet-based self-management intervention on patient empowerment, their management of the disease, and, ultimately, health outcomes. Methods A total of 51 patients were recruited through their health care providers and randomly assigned to either an experimental group with full access to the Internet-based intervention or a control group that was denied access to the interactive sections and knew nothing thereof. The intervention took 8 weeks. A baseline, a mid-term after 4 weeks, and a final assessment after 8 weeks measured patient empowerment, physical exercise, medication misuse, and pain burden. Results All patients completed the study. Overall, the intervention had a moderate effect (F 1.52=2.83, P=.03, η2=0.30, d=0.55). Compared to the control group, the availability of interactive sections significantly increased patient empowerment (midterm assessment: mean difference=+1.2, P=.03, d=0.63; final assessment: mean difference=+0.8, P=.09, d=0.44) and reduced medication misuse (midterm assessment: mean difference=−1.5, P=.04, d=0.28; final assessment: mean difference=−1.6, P=.03, d=−0.55) in the intervention group. Both the frequency of physical exercise and pain burden decreased, but to equal measures in both groups. Conclusions Results suggest that interactive sections as part of Internet-based interventions can positively alter patients’ feelings of empowerment and help prevent medication misuse. Detrimental effects were not observed. Trial Registration ClinicalTrials.gov: NCT02114788; http://www.clinicaltrials.gov/ct2/show/NCT02114788 (Archived by WebCite at http://www.webcitation.org/6ROXYVoPR).

Ultrasonography of haemophilic arthropathy

Summary.  Imaging is an essential tool for evaluation and monitoring of haemophilic arthropathy. Ultrasonography is increasingly used for joint assessment, due to its great sensitivity for soft tissue and relatively low cost. To assess the joint status and the role of ultrasonography in routine diagnosis and monitoring of joint disease in cohort haemophilic patients. Findings of patients with haemophilia, who routinely underwent ultrasonography were retrospectively evaluated to assess their joint status and the role of ultrasonography in routine diagnosis and monitoring of joint disease. Out of 325 joints examined (115 ankles, 210 knees), ultrasonography identified damages in 50% of ankles and 33% of knees in overall 111 patients, aged 7–80 years (median = 29 years). Synovial hypertrophy and cartilage abnormalities were the most frequent observations (88% and 76% in affected knees, respectively). Pristine joints were more frequently found in patients on primary prophylaxis, young age or no bleeding in the year prior to examination. Furthermore, no concordance was found between presence of joint changes at ultrasonography, and clinical joint status. Ultrasonography was shown to be able to detect joint damage involving soft tissues and bone surface. Its use might allow frequent monitoring of patients with haemophilia and early detection of arthropathy. For these reasons it might represent a valid tool in the routine management of haemophilia.

Causes, coping, and culture: a comparative survey study on representation of back pain in three Swiss language regions.

Introduction This study intends to contribute to a research tradition that asks how causal attributions of illnesses affect coping behavior. Causal attributions are understood as the most important element of illness representations and coping as a means to preserve quality of life. The issue is applied to a condition so far often neglected in research on illness representations–back pain–and a third concept is added to the picture: culture. Aim The aim of this study is (a) to explore the causal factors to which persons with back pain attribute the further course of their illness, (b) to find out whether the attributed causes are predictors of coping maxims, and (c) to find out whether cultural factors affect attributions and coping and moderate the relationship between the two. Methods A total of 1259 gainfully employed or self-employed persons with recent episodes of back pain were recruited in the three language regions of Switzerland. They were asked to complete a structured online interview, measuring among many other variables attributed causes, coping maxims, and affiliation to one of the Swiss micro-cultures (German-, French- or Italian-speaking). Results Attributed causes of the illness that can be influenced by a patient go along with more active coping styles. Cultural affiliation impacts on coping maxims independently, but culture moderates the relationship of attributed causes and coping maxims only in two of twenty possible cases. Implications The results show that cultural differences can be analytically incorporated in the models of illness representations. Results may help to improve healthcare providers’ communication with patients and plan public health campaigns. The approach to micro-cultural differences and the substantive relationships between alterability of causes and activity in coping may help the further development of models of illness representations.

The Representation of Risk in Routine Medical Experience: What Actions for Contemporary Health Policy?

Background The comprehension of appropriate information about illnesses and treatments, can have beneficial effects on patients’ satisfaction and on important health outcomes. However, it is questionable whether people are able to understand risk properly. Aim To describe patients’ representation of risk in common medical experiences by linking such a representation to the concept of trust. A further goal was to test whether the representation of risk in the medical domain is associated to the level of expertise. The third goal was to verify whether socio-demographic differences influence the representation of risk. Methods Eighty voluntary participants from 6 health-centers in northern Italy were enrolled to conduct a semi-structured interview which included demographic questions, term-associations about risk representation, closed and open questions about attitudes and perception of risk in the medical context, as well as about medical expertise and trust. Results The results showed that people do not have in mind a scientific definition of risk in medicine. Risk is seen as a synonym for surgery and disease and it is often confused with fear. However, general knowledge of medical matters helps people to have a better health management through risk identification and risk information, adoption of careful behaviors and tendency to have a critical view about safety and medical news. Finally, trust proved to be an important variable in risk representation and risk and trust were correlated positively. Conclusions People must receive appropriate information about the risks and benefits of treatment, in a form that they can understand and apply to their own circumstances. Moreover, contemporary health policy should empower patients to adopt an active self-care attitude. Methodologies to enhance people’s decision-making outcomes based on better risk communication should be improved in order to enable low literacy population as well elderly people to better understand their treatment and associated risk.

Linking quality-of-life measures using the International Classification of Functioning, Disability and Health and the International Classification of Functioning, Disability and Health-Children and Youth Version in chronic health conditions: the example of young people with hemophilia.

ObjectiveThis study aimed to measure the health and functioning of children with hemophilia in Europe using the International Classification of Functioning, Disability and Health (ICF) and the International Classification of Functioning, Disability and Health–Children and Youth Version (ICF-CY) as a frame of reference and items from health-related quality-of-life instruments as a measurement tool within a European data set. DesignBased on the results of linkage of items from the hemophilia-specific health-related quality-of-life questionnaire for children and adolescents to ICF/ICF-CY, the categories most relevant for the description of health and functioning of children with hemophilia were identified for each domain of the ICF/ICF-CY. Using data from the European Study of Clinical, Health, Economic, and Quality-of-Life Outcomes of Hemophilia treatment on 446 children, the frequency of impairments in body structures and body functions, restrictions in activities and participation, and barriers in contextual factors were calculated. ResultsIn general, the frequency of impairments, restrictions, and barriers was low in the studied population of children with hemophilia. However, the level of restriction as well as barriers was higher than the level of impairments. Older children, children receiving on-demand treatment, and children with severe hemophilia tended to have more problems compared with younger children, children receiving prophylaxis treatment, and children with mild to moderate hemophilia. ConclusionsUsing items from quality-of-life instruments with the ICF-CY as a frame of reference proved to be a useful approach for the assessment of health and functioning in children with hemophilia.

The choice dilemma in chronic hematological conditions: Why choosing is not only a medical issue? A psycho-cognitive perspective

Research in cognitive psychology focused on risk perception and decision making was shown to facilitate treatment choice and patients satisfaction with decision in a number of medical conditions, increasing perceived alliance between patient and physician, and adherence to treatment. However, this aspect has been mostly neglected in the literature investigating choice of treatment for chronic hematological conditions. In this paper, a patient centered model and a shared decision making (SDM) approach to treatment switch in chronic hematological conditions, in particular chronic myeloid leukemia, atrial fibrillation, and β-thalassemia is proposed. These pathologies have a series of implications requiring important decisions about new available treatments. Although new generation treatments may provide a significant improvement in patients health and health-related quality of life (HrQoL), a significant percentage of them is uncertain about or refuse treatment switch, even when strongly suggested by healthcare guidelines. Possible cognitive and emotional factors which may influence decision making in this field and may prevent appropriate risk-and-benefits evaluation of new treatment approaches are reviewed. Possible adaptive strategies to improve quality of care, patient participation, adherence to treatment and final satisfaction are proposed, and implications relatively to new treatment options available are discussed.

Hyperinsulinemia induced by highly active antiretroviral therapy in an adolescent with polycystic ovary syndrome who was infected with human immunodeficiency virus

OBJECTIVE To report a case of polycystic ovary syndrome (PCOS) in an adolescent infected with human immunodeficiency virus (HIV) and lipodystrophy whose insulin resistance was induced by highly active antiretroviral therapy (HAART). DESIGN Case report. SETTING Academic department of pediatrics. PATIENT(S) A 14-year-old vertically HIV-infected adolescent receiving HAART. INTERVENTION(S) Clinical observation, metabolic and endocrinologic assessment, imaging of ovaries by ultrasound, and of intra-abdominal adipose tissue content by magnetic resonance imaging. MAIN OUTCOME MEASURE(S) Lipodystrophy, insulin resistance, PCOS. RESULT(S) After 34 months of HAART this adolescent showed lipodystrophy with central obesity and insulin resistance after oral glucose tolerance test (OGTT). Ovaries were normal at ultrasonography. After 56 weeks of HAART, the adolescent showed more severe evidence of lipodystrophy and insulin resistance, and she developed acne, hirsutism, and amenorrhea. The following hormone levels were elevated: FSH 5.9 mUI/mL, LH 15.4 mUI/mL, LH/FSH ratio >2.5, free T 5.6 pg/mL, DHEAS 2,070 ng/mL, androstenedione (A) 3.42 ng/mL; whereas 17-beta-E(2), P, PRL, and free T(4) values were within the normal range. Ultrasonography demonstrated a typical polycystic echographic architecture of the ovaries. CONCLUSION(S) Lipodystrophy and insulin resistance are well-recognized side effects of HAART. Polycystic ovary syndrome might be an additional side effect secondary to insulin resistance and lipodystrophy.

A Cohort Pilot Study on HIV-Associated Neuropsychological Impairments in Hemophilia Patients.

Despite advances in the management of HIV infection with the introduction of combination antiretroviral therapy, it is well known that HIV can directly infect the central nervous system and, as a result of such infection, neuropsychological impairments can be manifested. In this study, we tried to determine whether seropositivity was associated with a poor neuropsychological performance in patients with hemophilia and HIV. Such a cohort of patients is very often underrepresented and understudied in the HIV literature. To amend such a gap, we carried out an extensive neuropsychological evaluation on these patients, and compared their performance with that of a group of seronegative hemophilia patients. The results revealed that HIV infection in HIV-seropositive (HIV+) hemophilia patients was associated with deficits in attention, short-term memory, abstraction, and visual recognition. Such results are still preliminary and explorative due to the small cohort of patients enrolled. However, the results do seem to have some important implications for day-to-day functioning, as the level of impairment detected may cause difficulties in completing common everyday tasks such as maintaining adherence to complex medication regimens or maintaining social life activities. Continued research into the mechanisms related to HIV and neurocognitive dysfunction may provide targets for interventions that could have meaningful consequences in the real world for HIV hemophilia patients.

Safety and effectiveness of raltegravir in patients with haemophilia and anti‐HIV multidrug resistance

Summary.  Highly active antiretroviral therapy (HAART) of HIV+ patients with haemophilia poses specific questions on safety and effectiveness because of long‐lasting HIV infection, multidrug resistance, concomitant chronic liver disease and bleeding risk. Raltegravir belongs to a new class of drugs that inhibits HIV integrase and is known to have a good effectiveness and safety profile. The aim of this study was to evaluate safety and effectiveness of HAART with raltegravir in patients with haemophilia. HIV+ patients with haemophilia treated with raltegravir for ≥6 months were included in this retrospective study. Safety criteria were: occurrence of any adverse event, unexpected blood test abnormalities and increased consumption of coagulation factors. Effectiveness criteria were: no disease progression, viral load <40 HIV‐RNA copies mL−1 and increased or stable CD3+ CD4+ cell count above 200 cells cmm−1. Seven patients with HCV co‐infection underwent treatment with raltegravir for a median of 20 months (min–max: 7–30). Before starting treatment with raltegravir, three patients had CD3+ CD4+ cell counts <200 cells cmm−1. The median viral load was 7547 copies mL−1 (min–max: <40–37 807). During treatment, no new sign of disease progression was observed. All patients showed suppression of viral replication (<40 HIV‐RNA copies mL−1). CD3+ CD4+ cell counts showed a median increase of 152 cells cmm−1 (min–max: 40–525). Two patients suffered from peripheral neuropathy, which was deemed as possibly associated with raltegravir. There was no evidence of increased bleeding frequency, modification of bleeding sites and lack of response to replacement therapy. Raltegravir‐based HAART appeared to be effective and generally well‐tolerated in patients with haemophilia, and it might represent a useful option in these patients.