Motor neuron disease

1) internet platform ALSHome.de
Self-assessment of symptom progression and quality of life in chronic diseases is of increasing importance in clinical research and specialised outpatient healthcare. Against this background, we developed the Internet portal www.ALShome.de which provides online access to the ALS Functional Rating Scale (ALSFRSr) and other established self-assessment questionnaires. In home care, an internet-based self-assessment is a possible perspective for the monitoring of ALS-associated symptoms. www.ALShome.de was created as a secure and closed Internet portal for patients. The application was developed in c-sharp (c#) with the persistent data storage being realized via an MS-SQL database throughout. Data get captured by generic questionnaires, are visualised on the website and administrated via a content management system. Prospective, controlled and stratified studies  for self-assessment of ALS-associated symptoms are conducted including more than 300 patients. The web-based self-assessment of ALS symptoms in a home care environment complements the well-established application of the ALSFRSr in outpatient departments. Current studies were able to demonstrate the medical, logistical and technical feasibility. The low drop out rate expresses the high acceptance of this online self-assessment tool among ALS patients. On-going investigations support the hypothesis that innovative elements of self-management perspectively gain significant relevance in outpatient care.

2) Internet-Based Assessment of Appetite
80% of ALS patients suffer from an undesirable loss of weight over the course of the disease. Underlying causes are malnutrition, cachexia as well as a multifactorial reduction of appetite.  The established ALS Functional Rating Scale, ALS-FRSr, records symptoms of dysphagia and manual deficits upon intake of food. The loss of appetite as a therapeutically relevant cause of undesirable weight loss is not reflected in the  ALS-FRSr. In a controlled study, we examine for the first time the symptoms of loss of appetite by means of a specific self-assessment score. A questionnaire is generated via the front-end of a web server featuring a specially secured operating system. Patients have access to a protected online portal via the URL www.ALS-home.de. The internet-based self-assessment is realised using the Council on Nutrition Appetite Questionnaire (CNAQ). Early ascertainment of indications for nutrition management  including ecotrophological consultation, supplementary nutrition and pharmacotherapy represent essential objectives in the treatment of undesirable weight loss.

3) Internet-Based Assessment of Dyspnoea
Respiratory insufficiency is a prognosis determining syndrome and a crucial focus in palliative care of ALS patients. Respiratory insufficiency is the subject of three arrays of questions (items) on the established self-assessment questionnaire ALS Functioning Rating Scale. One of the main limitations of the ALSFRSr is the lack of differentiation between neuromuscular hypoventilation and secretory pharyngeal and bronchial obstruction which in itself represents an independent risk factor for ALS-associated dyspnoea.  We present a study aimed at the obtainment of subjective assessments of different aspects of dispnoea performed by patients themselves. In a prospective, controlled and stratified study, patients conduct a self-assessment of dyspnoea. They are given access to the Internet portal www.ALShome.de. To evaluate dyspnoea in ALS we employed the Cancer Dyspnoea Scale (CDS) which is an established tool in oncological indications. The hypothesis of this examination is based on the possibility of a generic use of the CDS in ALS as it applicable in independence of a certain diagnosis. The symptom-oriented self-assessment of dyspnoea complements the established ALS Functioning Rating Score (ALSFRSr). In contrast to the ALSFRSr, the CDS focuses on the multi-dimensional subjective experience of dyspnoea. The early detection of dyspnoea by means of online self-assessment is of clinical relevance for the timely identification of indications for non-invasive ventilation or palliative medicamentous pharmacotherapy.

4) A pilot study of anakinra in ALS
Anakinra (ANA) is a naturally occurring interleukin-1 receptor antagonist (IL-1RA) with anti-inflammatory properties. We have initiated a controlled, open-label pilot study to evaluate the safety and tolerability of ANA in ALS. A secondary objective is to evaluate the effects of ANA on clinical function. We administer an investigational therapy of ALS with a daily subcutaneous injection of Anakinra (ANA) based on its anti-inflammatory properties. The monitoring of safety and clinical picture is performed by means of a web-based platform (www.ALSHome.de). 20 patients are currently enrolled in a phase IIb-controlled, open-label pilot study to test the safety and tolerability of ANA. The patients are treated with a daily subcutaneous injection of 100 mg ANA over a 52-week treatment period. The primary aim of the trial is to determine whether treatment with ANA is safe and well-tolerated in combination with riluzole. The parameters measured are the total number of adverse events (AE), the intraindividual rate of functional decline using the ALS Functional Rating Scale-Revised (ALS-FRS-R) and vital capacity (VC). For this pilot study, the web-based platform ALSHome.de was customized and implemented. ALSHome.de comprises several modules that allow for the monitoring of side effects and self-evaluation using the ALS-FRS-R. The trial is designed as a feasibility study in preparation for a larger phase IIb/III trial of efficacy. EudraCT-Nummer: 2010-019218-26

Collaboration

Arturo Zychlinsky , Max Planck Institute of Infection Biology, Berlin

Funding

Bundesministerium für Bildung und Forschung
Air Berlin Fonds for ALS Research
Private Donations
Industry

top