Quality of life measurement in urticaria: Position statement of the European Academy of Dermatology and Venereology Task Forces on Quality of Life and Patient-Oriented Outcomes and Urticaria and Angioedema.

The European Academy of Dermatology and Venereology (EADV) Task Forces on quality of life (QoL) and patient-oriented outcomes and on urticaria and angioedema recommendations for the assessment of Health-related (HR) QoL in all patients with urticaria in research and practice are as follows: to use the DLQI for adults and the CDLQI for children as dermatology-specific and the CU-Q2oL as a disease-specific HRQoL instruments in urticaria; to use generic instruments to provide comparison of data on urticaria with non-dermatologic diseases, or to compare with healthy volunteers or the general population; to select validated HRQoL instruments with appropriate age limits; to present exact numeric data for HRQoL results; correct title of any HRQoL instrument should be used, along with its correct abbreviation and the reference to its original publication, where possible. The EADV TFs discourage the use of non-validated HRQoL instruments and modified HRQoL instruments that have not undergone standard validation.

Quality of life measurement in teledermatology. Position statement of the European Academy of Dermatology and Venereology Task Forces on Quality of Life and Patient Oriented Outcomes and Teledermatology.

Many events, including the COVID-19 pandemic, have accelerated the implementation of teledermatology pathways within dermatology departments and across healthcare organizations. Quality of Life (QoL) assessment in dermatology is also a rapidly developing field with a gradual shift from theory to practice. The purpose of this paper organized jointly by the European Academy of Dermatology and Venereology (EADV) Task Force (TF) on QoL and patient-oriented outcomes and the EADV TF on teledermatology is to present current knowledge about QoL assessment during the use of teledermatology approaches, including data on health-related (HR) QoL instruments used in teledermatology, comparison of influence of different treatment methods on HRQoL after face-to-face and teledermatology consultations and to make practical recommendations concerning the assessment of QoL in teledermatology. The EADV TFs made the following position statements: HRQoL assessment may be an important part in most of teledermatology activities; HRQoL assessment may be easily and effectively performed during teledermatology consultations. It is especially important to monitor HRQoL of patients with chronic skin diseases during lockdowns or in areas where it is difficult to reach a hospital for face-to-face consultation; regular assessment of HRQoL of patients with skin diseases during teledermatology consultations may help to monitor therapy efficacy and visualize individual patient's needs; we recommend the use of the DLQI in teledermatology, including the use of the DLQI app which is available in seven languages; it is important to develop apps for dermatology-specific HRQoL instruments for use in children (for example the CDLQI and InToDermQoL) and for disease-specific instruments.

Quality of life measurement in rosacea. Position statement of the European Academy of Dermatology and Venereology Task Forces on Quality of Life and Patient Oriented Outcomes and Acne, Rosacea and Hidradenitis Suppurativa.

The European Academy of Dermatology and Venereology (EADV) Task Forces (TFs) on Quality of Life (QoL) and Patient-Oriented Outcomes and Acne, Rosacea and Hidradenitis Suppurativa (ARHS) do not recommend the use of any generic instrument as a single method of Health Related (HR) QoL assessment in rosacea, except when comparing quimp (quality of life impairment) in rosacea patients with that in other non-dermatologic skin diseases and/or healthy controls. The EADV TFs on QoL and Patient-Oriented Outcomes and ARHS recommend the use of the dermatology-specific HRQoL instrument the Dermatology Life Quality Index (DLQI) and the rosacea-specific HRQoL instrument RosaQoL in rosacea patients. The DLQI minimal clinically important difference may be used as a marker of clinical efficacy of the treatment and DLQI score banding of 0 or 1 corresponding to no effect on patients' HRQoL could be an important treatment goal. This information may be added to consensuses and guidelines for rosacea.

Quality of life measurement in alopecia areata. Position statement of the European Academy of Dermatology and Venereology Task Force on Quality of Life and Patient Oriented Outcomes.

New treatment options may lead to an increased interest in using reliable and sensitive instruments to assess health-related quality of life in people with alopecia areata (AA). The purpose of this paper is to present current knowledge about quality of life assessment in AA. The dermatology-specific Dermatology Life Quality Index (DLQI) was the most widely reported health-related quality of life instrument used in AA. Three AA-specific (Alopecia Areata Symptom Impact Scale, Alopecia Areata Quality of Life Index and Alopecia Areata Patients' Quality of Life) and three hair disease-specific instruments (Hairdex, Scalpdex and 'hair-specific Skindex-29') were identified with a range of content and validation characteristics: there is little evidence yet of the actual use of these measures in AA. Scalpdex is the best-validated hair disease-specific instrument. Further extensive validation is needed for all of the AA-specific instruments. The European Academy of Dermatology and Venereology Task Force on Quality of Life and Patient Oriented Outcomes recommends the use of the dermatology-specific DLQI questionnaire, hair disease-specific Scalpdex and the alopecia areata-specific instruments the Alopecia Areata Symptom Impact Scale or Alopecia Areata Quality of Life Index, despite the limited experience of their use. We hope that new treatment methods will be able to improve both clinical signs and health-related quality of life in patients with AA. In order to assess the outcomes of trials on these new treatment methods, it would be helpful when further development and validation of AA-specific instruments is being encouraged and also conducted.

Hyperhidrosis Quality of Life Index (HidroQoL©): further validation and clinical application in patients with axillary hyperhidrosis using data from a phase III randomized controlled trial.

BACKGROUND: The Hyperhidrosis Quality of Life Index (HidroQoL©) is a validated patient-reported outcome measure capturing the quality of life of people affected by hyperhidrosis. OBJECTIVES: We aimed to extend the validity evidence to physician-confirmed diagnosis of primary axillary hyperhidrosis. METHODS: Data from a phase III randomized placebo-controlled clinical trial were used (n = 171). Confirmatory factor analysis was carried out to confirm the a priori two-factor structure of the HidroQoL. Internal consistency was assessed using Cronbach's α. Intraclass correlation coefficients (ICCs) were calculated to evaluate test-retest reliability after days -7 to -4. Convergent validity was assessed using correlations with the Dermatology Life Quality Index (DLQI), the Hyperhidrosis Disease Severity Scale (HDSS) and gravimetric sweat production. Known groups were analysed to evaluate discriminative validity. Responsiveness after 29 days was assessed and minimal important difference (MID) values were calculated using both anchor- and distribution-based approaches. All analyses were carried out for total HidroQoL and its two domains. RESULTS: The two-factor structure of the HidroQoL was confirmed. Internal consistency and test-retest reliability were strong (Cronbach's α 0·81-0·90; ICCs 0·89-0·93). Correlations with other outcome measures were in line with a priori hypotheses. The HidroQoL discriminated between different severity groups (P ≤ 0·001) and showed sensitivity to change towards improvement (P < 0·001). An MID value of 4 is proposed for the total scale. CONCLUSIONS: This study supports excellent measurement properties including clinical applicability of the HidroQoL in primary axillary hyperhidrosis and suggests a MID of 4 be applied to clinical trial data.

'The psychosocial burden of alopecia areata and androgenetica': a cross-sectional multicentre study among dermatological out-patients in 13 European countries.

BACKGROUND: Hair diseases play an important burden on patients' lives, causing significant emotional and psychosocial distress. However, the impairment due to different hair conditions, such as alopecia areata (AA) and androgenetic alopecia (AGA), has rarely been compared. OBJECTIVE: The aim of this study was to assess the psychological burden of subgroups of patients with different hair diseases and to compare them to a healthy population. METHODS: In this study, we analysed a subgroup of patients with hair diseases from patients of a large multicentre study including 3635 dermatological patients and 1359 controls from 13 European countries. In the subgroup of patients with hair diseases, we analysed the socio-demographic characteristics, the stress level, and the impact of hair diseases on quality of life (QoL), anxiety, and depression and we compared them among patients with AA, AGA and healthy controls. RESULTS: The study population included 115 patients (77% women, 23% men) with hair diseases, 37 of whom with AA and 20 with AGA. Patients with hair diseases had a lower education level than healthy controls (medium educational level: 43% vs. 28%). Overall, 41% of the patients reported stressful life events during the last 6 months compared with 31% of the controls. Patients with the same age, sex, depression level and comorbidities had a worse QoL when suffering from AA than from AGA (Mean Dermatology Life Quality Index score: 5.8 vs. 2.5). CONCLUSION: Patients with hair diseases are more anxious, depressed and have a lower QoL than controls.

Association of IL-12 and TNF-α Polymorphisms with Graft-Versus-Host Disease in Allogeneic Hematopoietic Stem Cell Transplant Recipients.

BACKGROUND: Cytokines are important factors determining the outcome of transplantation. The host ability in cytokine production may be affected by cytokine genes polymorphisms. OBJECTIVE: To investigate the effect of IL-12 and TNF-α gene polymorphisms on outcome of hematopoietic stem cell transplantation. METHODS: 90 bone marrow transplant recipients were included in this study. 30 (33%) of 90 recipients experienced graft-versus-host disease (GVHD). IL-12 and TNF-α gene polymorphisms were evaluated by PCR-RFLP and ARMS-PCR method, respectively. RESULTS: No significant difference in the distribution of IL-12 (rs3212227 +1188 A/C) and TNF-α (rs 1800629 -308 G/A) genotypes and alleles was observed between those with and without GVHD. There was no significant association between the distribution of genotypes and the recipient sex. CONCLUSION: IL-12 (rs3212227 +1188 A/C) and TNF-α (rs 1800629-308 G/A) genotypes and alleles were not risk factors for development of GVHD.

The self-assessed psychological comorbidities of prurigo in European patients: a multicentre study in 13 countries.

BACKGROUND: Prurigo is defined by the presence of chronic pruritus and multiple localized or generalized pruriginous lesions. OBJECTIVE: The aim of this study was to assess the psychological burden of prurigo in patients of European countries. METHODS: In this multicentre European study, 3635 general dermatology outpatients and 1359 controls were included. Socio-demographic data and answers to questionnaires (regarding quality of life, general health, anxiety and depression and suicidal ideation) were collected. RESULTS: There were 27 patients with prurigo; of these, 63% were men, and the mean age was 58.6 years. Among patients with prurigo, 10 of 27 (37%) suffered from anxiety and 8 of 27 (29%) from depression. Suicidal ideation was reported in 5 of 27 (19%) patients, and for four of these five patients, suicidal ideation was related to their skin disease. These frequencies were higher in the 10 commonest dermatological diseases (including psoriasis, atopic dermatitis and leg ulcers). The impact on quality of life was severe, with a mean Dermatologic Life Quality Index (DLQI) of 12.4, with an extreme impact on quality of life for 23% of patients and a very large impact for 27% of patients. CONCLUSION: The psychological comorbidities of prurigo are common, greater than those of other skin diseases, and their impact on quality of life is significant. Thus, it is important to study this condition and to find new treatments.

Effects of replacing rye-grass (Lolium spp.) hay by banana (Musa acuminata L.) by-products on feed intake, growth, and feed conversion rate of Canary hair sheep breed (Pelibuey) lambs.

The objective of this study was to evaluate the effects of replacing rye-grass (Lolium spp.) hay with banana (Musa acuminata L.) by-products on feed intake, average daily gain (ADG), and feed conversion rate (FCR) in Pelibuey (hair sheep breed) lambs. The lambs (n = 22; initial BW = 14.8 ± 2.5 kg) were individually housed and fed on two different types of diet for 58 days: the first group (experimental diet) received as forage a mixture of fresh banana by-products composed of leaves and pseudostem. The second group received commercial rye-grass hay (conventional diet) as a fiber source. Both groups received a supplementary commercial concentrate food. The total daily forage intake and FCR were lower (P < 0.001) in animals fed on the conventional diet (rye-grass hay) than animals fed on the experimental diet (banana by-products). However, there were no significant differences in total dry matter intake, total digestible energy intake, total body weight gain, and ADG between the two groups. The results obtained in this study suggest that banana by-products could be used as alternative forage for Pelibuey lambs raised in subtropical conditions.

Dermatologists across Europe underestimate depression and anxiety: results from 3635 dermatological consultations.

BACKGROUND: It was recently demonstrated that a significant number of patients with common skin diseases across Europe are clinically depressed and anxious. Studies have shown that physicians not trained as psychiatrists underdiagnose depression. This has not been explored among dermatologists. OBJECTIVES: To estimate the concordance between clinical assessment of depression and anxiety by a dermatologist and assessment with the Hospital Anxiety and Depression Scale (HADS). METHODS: The study was an observational cross-sectional multicentre study of prevalent cases of skin diseases in 13 countries in Europe. Consecutive patients were recruited in outpatient clinics and filled in questionnaires prior to clinical examination by a dermatologist who reported any diagnosis of skin disease and signs of mood disorders. RESULTS: Analysis of the 3635 consultations showed that the agreement between dermatologist and HADS was poor to fair (lower than 0·4) for all diagnosis categories. The true-positive rate (represented by the percentage of dermatologists recognizing signs of depression or anxiety in patients with depression or anxiety as defined by a HADS value ≥ 11) was 44·0% for depression and 35·6% for anxiety. The true negative rate (represented by the percentage of dermatologists not detecting signs of depression or anxiety in non-depressed or non-anxious patients defined by HADS-value < 11) was 88.8% for depression and 85.7% for anxiety. CONCLUSIONS: Dermatologists in Europe tend to underestimate mood disorders. The results suggest that further training for dermatologists to improve their skills in diagnosing depression and anxiety might be appropriate. When present, the psychological suffering of patients with dermatological conditions needs to be addressed.

Measuring the impact of dermatological conditions on family and caregivers: a review of dermatology-specific instruments.

The patient is the centre of a web of relationships, and the impact of his/her disease on family members and caregivers must be taken into account. The aim of this study was to identify the specific instruments that measure the impact of a dermatological disease on the quality of life (QoL) of family members, by performing a systematic search of the literature. Fifteen papers were identified, describing the creation and validation of nine instruments. Four of them concerned atopic dermatitis (Dermatitis Family Index, DFI; Parents' Index QoL Atopic Dermatitis, PiQoL-AD; QoL in primary caregivers of children with atopic dermatitis, QPCAD; Childhood Atopic Dermatitis Impact Scale, CADIS), two measured the impact of psoriasis in family members (Psoriasis Family Index, PFI; FamilyPso), one the impact of epidermolysis bullosa (Epidermolysis Bullosa Burden of Disease, EB-BoD), one of ichthyosis (Family Burden Ichthyosis, FBI), and one was generic for dermatological conditions (Family Dermatology Life Quality Index, FDLQI). The European Academy of Dermatology and Venereology quality of life taskforce recommends that the impact of a skin disease on family and caregivers should be measured as part of any thorough evaluation of the burden of a disease. Guidelines are given to choose the most appropriate instruments.

Understanding clinician influences and patient perspectives on outpatient discharge decisions: a qualitative study.

OBJECTIVE: To observe the influences on clinicians when discharging patients, to explore patients' perspectives concerning their discharge or follow-up decision and to identify what patients think is important for clinicians to consider when taking a discharge decision. DESIGN: Qualitative study involving observations of consultations and semistructured interviews with outpatients. SETTING: National Health Service outpatient clinics at a university hospital secondary referral centre. PARTICIPANTS: 64 consultations were observed followed by 56 interviews with patients aged over 18 years. MAIN OUTCOME MEASURE: Analysis of patients' perspectives and expectations concerning whether or not they were discharged. RESULTS: 25 types of influences were observed to be influencing the discharge decision process. All 31 discharged patients appeared to accept the clinicians' decision; however, 10 (22%) of those patients later expressed disappointment. Patients' discontent was due to perceived clinicians' uncertainty in diagnosis (patients mentioning=2), poor acceptance of the diagnosis (2), disease not 'cured' (4), differing perception on medical needs (2), lack of concern for job demands (1), felt uninvolved in the decision-making (4), feeling rushed (3), prolonged open appointment (2), pushed to seek private care due to healthcare budget constraints (2), language barrier (1) and not keen to continue follow-up with general practitioner (2). Patients were happy when there was certainty of the diagnosis (19), clear treatment plan (16), advised on treatment side effects (7), given a contact number if symptoms recurred (4), considering their travelling and job demands (3). CONCLUSIONS: This study highlights the importance of accurately perceiving patients' perspectives in ensuring the appropriateness of outpatient discharge. There was a disparity between patients' and clinicians' perception on what was an appropriate discharge. This included discrepancies concerning diagnostic certainties, private healthcare as an alternative, need for easy reaccess and choice of words surrounding discharge. Medical education should include handling these issues.

Once-weekly prophylactic treatment vs. on-demand treatment with nonacog alfa in patients with moderately severe to severe haemophilia B.

INTRODUCTION: Limited data are available on optimal prophylaxis regimens of factor IX (FIX) replacements for patients with haemophilia B. AIM: This multicentre, open-label study evaluated the efficacy and safety of once-weekly prophylaxis with nonacog alfa compared with on-demand treatment in adolescent and adult patients. METHODS: Males aged 12-65 years with moderately severe to severe haemophilia B (FIX:C ≤ 2%) were eligible for enrolment. Patients received on-demand treatment for 26 weeks, followed by once-weekly prophylaxis of 100 IU kg(-1) for 52 weeks. The primary efficacy end point was the annualized bleeding rate (ABR). Secondary end points included response to on-demand treatment, the number of infusions used to treat bleeding events, and the incidence of less-than-expected therapeutic effect (LETE). FIX:C was measured on day 1 and at weeks 26 and 78. RESULTS: Mean (±SD) ABR was lower during prophylaxis vs. on-demand treatment [3.6 (±4.6) vs. 32.9 (±17.4) events, respectively; P < 0.0001]. The majority (88.4%) of bleeding events had excellent or good responses upon the first infusion; 82.1% of events responded to the first infusion. No incident of LETE occurred. No thrombotic events or FIX inhibitors were reported. Eight of 17 FIX:C approximately 1 week after dosing were >2 IU dL(-1) (min-max of 2.13-10.39 IU dL(-1) ). CONCLUSIONS: Once-weekly prophylaxis of 100 IU kg(-1) was associated with lower ABR compared with on-demand treatment in adolescents and adults with moderately severe to severe haemophilia B. Once-weekly prophylaxis was well tolerated, with a similar safety profile as that reported during the on-demand treatment period. Residual FIX:C may be supportive of effectiveness.

First prospective report on immune tolerance in poor risk haemophilia A inhibitor patients with a single factor VIII/von Willebrand factor concentrate in an observational immune tolerance induction study.

INTRODUCTION/BACKGROUND: Development of neutralizing inhibitors against factor VIII (FVIII) is a major complication of haemophilia A treatment. AIM: The ongoing, international, open-label, uncontrolled, observational immune tolerance induction (ObsITI) study evaluates ITI, the standard of care in patients with inhibitors. PATIENTS/METHODS: Forty-eight prospective patients in this interim analysis received a single plasma-derived, von Willebrand factor-stabilized, FVIII concentrate (pdFVIII/VWF) for ITI. According to recommended Bonn protocol, 'low responders' at ITI start (<5 BU) received 50-100 IU FVIII kg(-1) daily, or every other day; 'high responders' (≥5 BU) received 100 IU FVIII kg(-1) every 12 h. RESULTS: Forty of 48 patients (83.3%), had at least one risk factor for poor ITI-prognosis at ITI start (i.e. age ≥7 years, >2 years since inhibitor diagnosis, inhibitor titre ≥10 BU at the start of ITI, or prior ITI failure). Nonetheless, 34 patients (70.8%) achieved complete success, 3 (6.3%) partial success, 1 (2.1%) partial response; ITI failed in 10 patients (20.8%), all with poor prognosis factors. All six low responders achieved complete success. ITI outcome was significantly associated with inhibitor titre level at ITI start (P = 0.0068), number of poor prognosis factors for ITI success (P = 0.0187), monthly bleeding rate during ITI (P = 0.0005) and peak inhibitor titre during ITI (P = 0.0007). Twenty-two of 35 high responder patients (62.9%) with ≥1 poor prognosis factor achieved complete success. CONCLUSION: Treatment with a single pdFVIII/VWF concentrate, mainly according to the Bonn protocol, resulted in a high ITI success rate in haemophilia A patients with inhibitors and poor prognosis for ITI success.

pH control in biological systems using calcium carbonate.

Due to its abundance, calcium carbonate (CaCO3) has high potentials as a source of alkalinity for biotechnological applications. The application of CaCO3 in biological systems as neutralizing agent is, however, limited due to potential difficulties in controlling the pH. The objective of the present study was to determine the dominant processes that control the pH in an acid-forming microbial process in the presence of CaCO3. To achieve that, a mathematical model was made with a minimum set of kinetically controlled and equilibrium reactions that was able to reproduce the experimental data of a batch fermentation experiment using finely powdered CaCO3. In the model, thermodynamic equilibrium was assumed for all speciation, complexation and precipitation reactions whereas, rate limited reactions were included for the biological fatty acid production, the mass transfer of CO2 from the liquid phase to the gas phase and the convective transport of CO2 out of the gas phase. The estimated pH-pattern strongly resembled the measured pH, suggesting that the chosen set of kinetically controlled and equilibrium reactions were establishing the experimental pH. A detailed analysis of the reaction system with the aid of the model revealed that the pH establishment was most sensitive to four factors: the mass transfer rate of CO2 to the gas phase, the biological acid production rate, the partial pressure of CO2 and the Ca(+2) concentration in the solution. Individual influences of these factors on the pH were investigated by extrapolating the model to a continuously stirred-tank reactor (CSTR) case. This case study indicates how the pH of a commonly used continuous biotechnological process could be manipulated and adjusted by altering these four factors. Achieving a better insight of the processes controlling the pH of a biological system using CaCO3 as its neutralizing agent can result in broader applications of CaCO3 in biotechnological industries.

New quantitative aPTT waveform analysis and its application in laboratory management of haemophilia A patients.

Diagnosis of haemophilia A is usually made by the measurement of factor VIII (FVIII) activity that allows categorization of the disease severity. However, tests that assess global haemostasis may better reflect clinical features and give additional clinically relevant information. The aim of this study was to develop a new quantitative activated partial thromboplastin time (aPTT) waveform analysis and compare it with FVIII activities to find out whether waveform parameters are superior determinants of clinical phenotype. A total of 81 haemophilia A patients divided into two groups (37 severe, 44 non-severe) were included in the study. The control group comprised 101 healthy male volunteers. Quantitative aPTT waveform analysis was performed with Actin FS on BCS (Siemens Healthcare Diagnostics, Marburg, Germany) using three parameters (DELTA, RATIO-1, RATIO-2) obtained from a single aPTT measurement with two evaluation modes. FVIII activities were measured by one-stage clotting and two-stage chromogenic assay. Statistically significant difference (P < 0.001) between control group and all haemophilia A patients, as well as between severe and non-severe haemophilia A patients was obtained for all quantitative waveform parameters. Our study revealed parameter DELTA as the best waveform parameter, showing significant correlation with FVIII activities and clinical parameters, and excellent performance for distinguishing between severe and non-severe haemophilia A patients (ROC analysis: sensitivity 97.3%, specificity 93.2%). The results obtained by new quantitative aPTT waveform analysis were superior to those obtained by standard laboratory methods. The simplicity and cost-benefit of the method make this approach a reasonable and promising tool for assessing coagulation in haemophilia A patients.

The dermatology outpatient discharge decision: understanding a critical but neglected process.

Discharge from dermatology outpatients is a critical endpoint of patient care. Despite this, there has been very little research concerning the discharge process and factors influencing the discharge decision. To identify the factors influencing discharge decisions, articles from 1970 to April 2013 were searched in MEDLINE via Ovid, CINAHL, PROQUEST and Google Scholar using the keywords 'patient discharge', 'discharge decision', 'factors influencing discharge', 'clinical decision making', 'discharge decision making', 'process of discharge decision', 'outpatient', 'follow up', 'skin disease' and 'dermatology'. Only articles describing outpatient discharge decisions were included. Seventeen outpatient discharge articles were identified, 12 from the U.K. (seven dermatology) and five from the U.S.A., Canada, Australia and Taiwan (all nondermatology). The main influences on outpatient discharge identified were diagnosis and disease severity, clinician's level of experience and perception, patient's preferences, patient's behaviour and quality of life. These influences affected the clinician's judgement on discharge decisions both in appropriate and in inappropriate ways. Little is known concerning discharge decision making in dermatology. Given the central importance of such decisions in the appropriate care of patients and the efficient running of any dermatology service, greater understanding of the influences on discharge decision making is needed. It is therefore critical for dermatologists to be aware of these influences and to ensure that decisions are taken only in the best interests of patients. Further research is required to inform the training of dermatologists on how to take the most appropriate discharge decisions.

Infants' Dermatitis Quality of Life Index: a decade of experience of validation and clinical application.

The Infants' Dermatitis Quality of Life Index (IDQoL) is a questionnaire completed by parents to assess the impact of atopic dermatitis on the quality of life of infants aged 0-3 years. The aim was to review all clinical and psychometric data on the use of the IDQoL from its inception in 2001 until November 2012, to serve as a single reference source. A literature search was carried out using Medline, PubMed, EBSCOhost, Science Direct and Google Scholar. Articles and abstracts that described the clinical use of the IDQoL and those that investigated its psychometric properties were included. Articles not in English were excluded. Fifty-one publications were identified, of which 46 fulfilled the inclusion criteria. IDQoL data relating to psychometric, descriptive and clinical practice research, drug trials and therapeutic interventions were extracted. The IDQoL has been translated into 21 languages and used in 18 countries, including two multinational studies. Thirty-one studies demonstrated its psychometric properties, such as test-retest reliability, internal consistency, validity, responsiveness to change and interpretability. No studies investigated dimensionality, carried out factor analysis or described the minimal clinically important difference of the tool. Eight studies used the IDQoL to assess the effectiveness of therapeutic interventions such as education programmes, consultations and wet-wrap therapy, while seven studies described the use of IDQoL in topical interventions. When deciding whether to use the IDQoL, researchers and clinicians should consider the validation data, the relevant comparative clinical data available and the potential burden on the respondents.

Results from a large multinational clinical trial (guardian™1) using prophylactic treatment with turoctocog alfa in adolescent and adult patients with severe haemophilia A: safety and efficacy.

Recombinant factor VIII (rFVIII) products provide a safe and efficacious replacement therapy for prophylaxis and treatment of bleeding episodes in patients with severe haemophilia A. This multinational, open-label, non-controlled trial investigated the safety and efficacy of turoctocog alfa, a new rFVIII product. The primary objective was to evaluate safety. A total of 150 patients (24 adolescents and 126 adults) with severe haemophilia A (FVIII activity ≤ 1%), with at least 150 exposure days (EDs) to any FVIII product and no history of inhibitors were enrolled, and 146 patients (97%) completed the trial. All patients received prophylaxis with turoctocog alfa for approximately 6 months and had a mean of 85 EDs during the trial. None of the patients developed FVIII inhibitors, there were no indications of early FVIII inhibitor development and no safety concerns were identified. A total of 225 adverse events were reported in 100 (67%) patients, with the most common being events associated with dosing procedures, headaches, and nasopharyngitis. A total of 499 bleeding episodes were reported during the trial, the majority (89%) were controlled with 1-2 infusions of turoctocog alfa. Based on patient reports, the success rate (defined as 'excellent' or 'good' haemostatic response) for treatment of bleeding episodes was 81%. The overall median annualized bleeding rate was 3.7 (interquartile range: 8.7) bleeds/patient/year. In conclusion, turoctocog alfa provides a new, safe and effective alternative for prophylaxis and treatment of bleeding episodes in patients with haemophilia A.