Development, validation, and initial results of the Angioedema Activity Score.

BACKGROUND: Recurrent angioedema (RecA) is a frequent clinical problem characterized by suddenly occurring cutaneous and/or mucosal swellings. Depending on their location, RecA may be painful, hindering, disfiguring, or even life-threatening. The assessment of disease activity in affected patients is important to guide treatment decisions. Currently, however, there is no standardized and validated outcome measure available to do so. OBJECTIVE: To develop and validate the first specific patient-reported outcome instrument to assess disease activity in RecA patients, the Angioedema Activity Score (AAS). METHODS: After a set of potential AAS items was developed, item evaluation and reduction were performed by means of impact analysis, factor analysis, regression analysis, and by checking for face validity. In addition, the items of the final AAS questionnaire were tested for their validity and reliability during a 12-week validation study. RESULTS: In total, data from 110 and 80 RecA patients were used during the AAS item evaluation and validation phase, respectively. The resulting AAS consisted of five items and was found to have a one-dimensional structure and excellent internal consistency. It correlated well with other measures of disease activity and quality-of-life impairment, thus demonstrating its convergent validity. In addition, the known-groups validity and test-retest reliability of the AAS were found to be good. CONCLUSIONS: The AAS is the first validated and reliable tool to determine disease activity in RecA patients, and it may serve as a valuable instrument in future clinical studies and routine patient care.

[Useful tools for documenting urticaria]. / Nützliche Instrumente für die Dokumentation bei Urtikaria.

Urticaria is one of the most common inflammatory skin diseases. Many patients suffer for many years of the subtypes of urticaria. Because of their symptoms, urticaria patients have a very low quality of life and are subject to comorbidities. A structured approach with validated evaluation tools helps (1) to diagnose the respective subtype of the disease, (2) to define the disease activity and (3) to analyze the impact on quality of life. By virtue of this, it is possible to adequately diagnose and manage urticaria patients without major investment of time or manpower. Tools to measure disease intensity (urticaria activity score, angioedema activity score) and questionnaires for quality of life in the cases of chronic urticaria and angioedema have been developed. Suspected diagnoses of comorbidity such as depression or anxiety can be confirmed by the appropriate questionnaire (Hamilton Anxiety Depression Scale [HADS]). In addition diagnostic instruments are helpful to define the intensity of the disease, especially in cases of inducible urticaria. All these forms, scores and questionnaires are easy to learn and employ in daily practice.

Development and construct validation of the angioedema quality of life questionnaire.

BACKGROUND: Recurrent angioedema is a frequent clinical problem characterized by unpredictably and rapidly occurring cutaneous and mucosal swellings. These swellings may be painful and/or disfiguring. Upper airway involvement can also lead to dyspnea and suffocation. Although the disease burden is high, there is currently no specific instrument to measure health-related quality of life (QoL) impairment. OBJECTIVE: To develop and validate the first symptom-specific tool to assess QoL impairment in recurrent angioedema patients, adhering to established methodological recommendations. METHODS: During the development phase, 29 questions (items) were generated. Subsequently, item reduction was performed by means of impact analysis and factor analysis as well as by checking for content and face validity. As a result, 17 items were selected and included in the final instrument, the Angioedema QoL Questionnaire (AE-QoL). AE-QoL was then tested for its validity, reliability, and influence factors. RESULTS: One hundred and ten angioedema patients took part in the validation of AE-QoL. AE-QoL was found to have a four-dimensional structure as well as a valid total score. All of its four domains (functioning, fatigue/mood, fears/shame, food) showed good levels of internal consistency with Cronbach's alpha > 0.8. Test-retesting revealed a good reliability of the instruments total score and domain scores. Gender as well as the patients' self-rated disease activity was found to be predictors of the AE-QoL total score. CONCLUSIONS: Angioedema Quality of Life Questionnaire is the first angioedema-specific QoL questionnaire. It is a short, valid and reliable instrument that may serve as a valuable tool in future clinical studies and in routine patient care.

[Systemic therapy for malignant melanoma]. / Medikamentöse Therapie des malignen Melanoms.

For decades dacarbazine was the standard in the therapy for metastatic melanoma even though response rates were low. In recent years multiple pharmacological approaches have led to new therapy options including immune modulators like anti-CTLA4 antibodies and kinase inhibitors of the MAPK signaling pathway that showed better response rates and increased overall survival. However, since immune modulators lead only in a small subgroup of patients to long-term responses and kinase inhibitors lose their function due to development of resistance after several months, continuation of clinical studies is strongly required. Classical chemotherapeutic drugs will remain a basic part of the therapy especially as combinations of different treatment options have to be focused on in order to achieve better long-term survival rates.

[Reaction to measles vaccine/vaccine-induced measles]. / Impfmasern/vakzineinduzierte Masern.

With a worldwide annual incidence of 31 million cases resulting in 614,000 fatalities in 2002, measles is a main cause of childhood death which could be prevented by vaccination. Since the introduction of immunization with attenuated measles virus vaccine, which led to a decrease in measles cases with a low incidence of 0.2/100,000 inhabitants in Germany in 2004, the population's awareness of the risks of measles has faded. Instead public interest has increasingly focussed on the possible but mostly harmless complications of vaccination. There is concern that the number of those not immunized will increase to such an extent that endemic outbreaks of measles will again occur.

[Successful use of allogeneic stem cell transplantation for treatment-refractory mycosis fungoides]. / Erfolgreicher Einsatz einer allogenen Stammzelltransplantation bei therapierefraktärer Mycosis fungoides.

Mycosis fungoides is the most common type of cutaneous T-cell lymphoma and characterized by a chronic progressive course spanning decades. The choice of treatment options should be tailored to the stage depending on the extent and aggressiveness of the disease and taking the individual situation of the patient into consideration. Long-term complete remissions can only be achieved in the early phase of the disease, while there is no therapy that results in a cure or long-term remission in advanced stages. In young patients with a treatment-refractory course of mycosis fungoides, allogeneic stem cell transplantation represents an important alternative option to manage the disease since complete clinical remission can be obtained even in advanced stages.

[Cutaneous larva migrans]. / Kutane Larva migrans.

As a result of the rise of mass tourism with increasingly cheap and exotic destinations, tropical diseases are becoming an increasingly important part of dermatology. Infection with cutaneous larva migrans is one of the most common "souvenirs" from the tropics. The disease is caused by the nematode infection with dog or cat hookworm parasites (Ancylostoma braziliense or A. caninum). The characteristic manifestation is a gyrated, serpiginous and in some cases vesicular erythema, which appears after penetration of the epidermis by the parasite and the subsequent intraepidermal migration of the larva. This is often accompanied by intense pruritus in the affected skin. The infection is usually found in those areas of the foot, calf or buttocks exposed by walking or sitting on sand. Although subjectively very unpleasant, the disease is self-limiting and resolves after several weeks; it is treated locally in most cases (e.g. using cryotherapy). The prognosis of the disease is excellent, but a prolonged course or complications have been observed, e.g. due to superinfection.