Twelve patients with severe intraventricular haemorrhage (IVH) underwent intraventricular thrombolysis with recombinant tissue plasminogen activator (rtPA). External ventricular drainage was performed in all patients within 24 hours of haemorrhage. Fibrinolytic therapy was started within 24 hours from the onset of symptoms in ten cases, and in two further cases after 48 hours and 5 days, respectively. Two to 5 mg of rtPA were injected via the ventricular catheter into one or both lateral ventricles. The injection was repeated at intervals ranging from 6 to 24 hours until CT scans demonstrated a substantial reduction of intraventricular blood. The total rtPA doses per patient ranged from 3 to 31 mg. CT scans showed a marked reduction of intraventricular blood and normalization of ventricular size within 24 to 48 hours from the beginning of the fibrinolytic therapy. Rapid reduction of elevated intracranial pressure by continuous diversion of cerebrospinal fluid could be achieved in all patients, because the ventricular catheters never became obstructed by clotted blood during the fibrinolytic therapy. During the period of treatment, the level of consciousness, as classified according to the Glasgow Coma Scale, improved from a mean value of 7 to 12. One fatal case of meningitis most probably due to the ventriculostomy was the only complication related to the treatment. This method of treatment might improve the prognosis in patients in whom a large intraventricular haematoma volume, ventricular dilatation, and impaired cerebrospinal fluid circulation are major determinants for the outcome.
Acquired aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are interrelated ultra-rare diseases. Quality of life (QoL) evaluation tools used in studies for AA and PNH are unspecific and designed for cancer patients (e.g., the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire, EORTC QLQ-C30). Given the complexity of AA and PNH, variation in symptoms and treatments, younger age of many patients, and the fact that AA and PNH are not classified as malignant diseases, it is likely that cancer-specific questionnaires are inappropriate. We generate an AA/PNH-specific QoL questionnaire (QLQ-AA/PNH), performed according to EORTC guidelines. QoL issues were obtained from the literature and interviews with patients and physicians (phase I), then ranked by patients and physicians. In phase II, items were created. Patients in more than 25 German and Swiss cities were interviewed face to face. In phase I, interviews of 19 patients and 8 physicians specialized in AA/PNH treatment resulted in 649 QoL issues; these were condensed to 175 and graded according to their importance by 30 patients and 14 physicians (phase II). Five physicians took part in phases I and II. Altogether, 97 issues were rated important. Twelve EORTC QLQ-C30 items were not rated important, while several new QoL aspects were brought up. Modifications in wording and phrasing led to two questionnaires with 77 items regarding general QoL aspects and 20 items regarding medical care. Important QoL aspects of PNH/AA patients are inappropriately captured with available QoL tools. Developing a new QoL questionnaire specific for this patient group is warranted.Electronic supplementary materialThe online version of this article (doi:10.1007/s00277-016-2867-8) contains supplementary material, which is available to authorized users.
To date, instruments to measure quality of life (QoL) specifically for patients with acquired aplastic anaemia (AA) and paroxysmal nocturnal haemoglobinuria (PNH) are lacking altogether. As a consequence, this issue is either underevaluated or alternatively, instruments originally designed for cancer patients are being used. We therefore started to systematically develop a AA/PNH-specific QoL (QLQ-AA/PNH) instrument in these ultra-rare diseases according to European Organisation for Research and Treatment of Cancer (EORTC) guidelines. While phases I and II of the process have previously been published, we now report on the resulting instrument (phase III of this process). As part of the phase III of the evaluation process, we approached patients through physicians, patient support groups, and patient conferences. After participants completed the preliminary questionnaire and reported socio-demographic data, they were interviewed in person or via phone with a debriefing interview to find out whether the items were relevant, easy to understand, and acceptable to patients and whether there was anything missing in the questionnaire. We hypothesised what items could be combined into a scale and calculated Cronbach’s alpha to define its preliminary internal consistency. After definition of a priori criteria to keep or delete items, a group of six experts met in person, discussed the results, and decided on in- or exclusion. A total of 48 patients were enrolled, 21 of those suffered from AA (44%), 13 from PNH (27%), and 14 from AA/PNH syndrome (29%). The median time to complete the 69 items was 10 min (range 5–20), mean time 11 min. The compliance criterion (> 95% completion) was fulfilled by 57 items. Twenty-three items were mentioned as especially relevant by ≥ 2% of the patients. Cronbach’s alpha of the hypothesised scales ranged from 0.63 (social support) to 0.92 (fear of progression and illness intrusiveness). Finally, 47 items were kept; 16 were deleted, and 5 were changed, while 1 item expanded. This resulted in 54 items in total. As no issues were mentioned to lacking by a minimum of five patients, no items were added to the questionnaire. After completion, the AA/PNH-QoL tool (QLQ-AA/PNH) was translated according to EORTC guidelines into English, French, and Italian. For patients with PNH and AA until now, the standard assessment for QoL was to use the EORTC Quality of Life Questionnaire (QLQ-C30) or the Functional Assessment of Chronic Illness Therapy Fatigue Instrument (FACIT-Fatigue). We herewith present a new instrument aimed to be better tailored to the needs of PNH and AA patients. The anticipated fourth development phase will be performed for psychometric validation; however, we already explored the internal consistency of the hypothesised scales and found the results to be very good. Hence, the new QLQ-AA/PNH with 54 items can be used in trials and clinical studies from now on, according to EORTC strategy even if the scoring algorithm at this point is preliminary and the QLQ-AA/PNH might change sl...
Introduction Acquired Aplastic Anemia (AA) and Paroxysmal Nocturnal Hemoglobinuria (PNH) are ultra-rare diseases with a yearly incidence 2 to 4 per million (AA) and of 1.3 to 2 per million (PNH. While much is known about pathophysiology and treatment of these interrelated diseases, less is known about patients (pts.) psycho-social issues. Quality of life (QoL) evaluation tools used in all studies for AA and PNH are rather unspecific and were initially designed for cancer patients (e.g. the European Organization of Research and Treatment (EORTC) QLQ-C30). Given the complexity of AA and PNH, the variation in symptoms and different treatment approaches (immunosuppression, bone marrow transplantation, complement inhibition or others and eventually sequences of all in some pts.), the often young age of the pts., and the fact that marrow failure syndromes are not classified as malignant diseases, it is likely that the cancer-specific questionnaires used so far are inappropriate to adequately assess the QoL and illness intrusiveness in these pts.Based on these considerations and funded by German patient support groups (Aplastische Anämie e.V., pnh-aa.info), we therefore initiated the development and validation of an AA/PNH-specific QoL-instrument (QLQ-AA/PNH). Methods Generation of a QLQ-AA/PNH was performed according to EORTC QoL group guidelines, i.e. after identification of QoL issues by literature review, a focus group of pts. and physicians were interviewed (phase I). After screening of documented interviews, QoL issues were generated and reworded in a preliminary questionnaire (phase II). In phase III the questionnaire with generated items was evaluated in a representative group of patients. Pts. were recruited through participating physicians, the internet and through word-of-mouth by other pts., pts. internet forums and pts. conferences. Individual pts. in more than 25 German and Swiss cities were visited and personally interviewed. Currently, psychometric validation of the final questionnaire is ongoing through patients included in the German PNH-registry. Eventually, the QLQ-AA/PNH will be further evaluated as part of a prospective clinical interventional trial performed within the European Group for Blood and Marrow Transplantation Working Party Aplastic Anemia (EBMT WP-AA). In addition, patients were asked to complete a questionnaire regarding their supportive care needs, (e. g. informations, support by medical staff, psychosocial counseling, patient support groups.) and potential iatrogenic problems (e. g. delay in diagnosis, appreciation of QoL problems by health care professionals). Results In phase I, 19 pts. and 8 physicians specialized in treatment of AA/PNH were interviewed. 649 resulting QoL issues were condensed to 175, which were then again graded according to their importance by 30 pts. (10 AA, 10 PNH, 10 PNH/AA), and 14 physicians (phase II). Altogether, 97 issues were rated important. Physicians rated fatigue the most important issue while pts. rated mutual trust between physicians and pts. as the most important QoL-aspect. 11/30 EORTC QLQ-C30 issues (e.g. loss of appetite, nausea, vomiting, constipation, diarrhea) were not mentioned or rated unimportant, while several new QoL aspects such as constant fear of infection and blood count variations, dependency on time-consuming therapies, and emotional strain through endlessness of disease burden were brought out. Modifications in wording and phrasing led to two questionnaires with 67 items regarding general QoL-aspects and further 20 items regarding medical care, which are currently being evaluated through interviews with 35 additional patients. The final questionnaire with no more than 40 items will then be validated. Conclusion A detailed summary of the relevant items or questions will be presented. Up to now important QoL-aspects of PNH/AA pts. are inappropriately captured or missed the with available EORTC-QoL-tools. Even in ultra-orphan diseases industry independent development of disease specific QoL-tools is feasible with support of pts. and pt. advocacy groups. Disclosures: No relevant conflicts of interest to declare.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
hi@scite.ai
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.