Publikationen

Rudolf Marx

ausgewählte Publikationen

Rudolf Marx1912 – 1990

Erforschung der Blutstillung als Lebensaufgabe

Wolfgang Schramm Schillerstr. 40, 80336 München W.Schramm@med.uni-muenchen.de

LMU Zentrum Seniorenstudium, München 18.12. 2013

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Started 25 years earlier than the first aPCCFEIBA

Grundlagenwerke

zur Hämostaseologie

Habilitationsschrift Rudolf Marx

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Wolfgang Schramm, Hrsg.: Chronologie der Hepatitis bei Hämophilie. München, 2003. ISBN-Nr. 3-927578-31-2



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Ausgewählte

Publikationen

With the aging population in Europe it is anticipated that the growing demand for blood products will not be met by the estimated supply. Therefore up-to-date recommendations for optimal administration of blood products in hemotherapy are needed. Ten years after the first meeting on optimal use of blood products at Wildbad Kreuth, Germany, a second symposium was organized to convene leading experts from the clinical, regulatory and economic perspective.  The aim was to re-evaluate the existing state of hemotherapy, identify areas where further studies are needed, and to provide up-dated recommendations. A preparatory survey by questionnaire concerning guidelines, quality management in clinical use of blood products, provision of products in the individual countries and re-evaluation of the 1999 Wildbad Kreuth recommendations was completed in advance. The second Kreuth Meeting in April 2009 was attended by 110 experts in transfusion medicine, regulators and regulatory authorities from 38 countries. By consensus, 20 new recommendations were adopted. Most of the 1999 recommendations were found to still be valid 10 years later. But their realization and implementation on the levels of clinical practice, regulatory authorities and health policy decision makers is still lagging behind leaving an important task to accomplish. The Kreuth initiative toward optimal use of blood products should continue. Berger K et al.: The Wildbad Kreuth initiative: European current practices and recommendations for optimal use of blood components. Biologicals.2011 May;39(3):189-93. doi: 10.1016/j.biologicals.2011.03.002. Epub 2011 Apr 23.
With the aging population in Europe it is anticipated that the growing demand for blood products will not be met by the estimated supply. Therefore up-to-date recommendations for optimal administration of blood products in hemotherapy are needed. Ten years after the first meeting on optimal use of blood products at Wildbad Kreuth, Germany, a second symposium was organized to convene leading experts from the clinical, regulatory and economic perspective.  The aim was to re-evaluate the existing state of hemotherapy, identify areas where further studies are needed, and to provide up-dated recommendations. A preparatory survey by questionnaire concerning guidelines, quality management in clinical use of blood products, provision of products in the individual countries and re-evaluation of the 1999 Wildbad Kreuth recommendations was completed in advance. The second Kreuth Meeting in April 2009 was attended by 110 experts in transfusion medicine, regulators and regulatory authorities from 38 countries. By consensus, 20 new recommendations were adopted. Most of the 1999 recommendations were found to still be valid 10 years later. But their realization and implementation on the levels of clinical practice, regulatory authorities and health policy decision makers is still lagging behind leaving an important task to accomplish. The Kreuth initiative toward optimal use of blood products should continue. Berger K et al.: The Wildbad Kreuth initiative: European current practices and recommendations for optimal use of blood components. Biologicals.2011 May;39(3):189-93. doi: 10.1016/j.biologicals.2011.03.002. Epub 2011 Apr 23.
This report summarizes recommendations relating to haemophilia therapy arising from discussions among experts from 36 European countries during the Kreuth III meeting in April 2013. To optimize the organization of haemophilia care nationally, it is recommended that a formal body be established in each country to include the relevant clinicians, national haemophilia patient organization, health ministry, paying authority and (if appropriate) regulatory authorities. The minimum factor VIII consumption level in a country should be 3 I.U. per capita. Decisions on whether to adopt a new product should not be based solely on cost. Prophylaxis for children with severe haemophilia is already recognized as the optimum therapy. Ongoing prophylaxis for individual adults should also be provided when required based on clinical decision making by the clinician in consultation with the patient. Children with inhibitors who have failed, or who are not suitable for, immune tolerance therapy should be offered prophylaxis with bypassing agents. Single factor concentrates should be used as therapy wherever possible in patients with rare bleeding disorders. Orphan drug designation for a factor concentrate should not be used to hinder the development, licencing and marketing of other products for the same condition which have demonstrably different protein modification or enhancement. Giangrande P et al.: Kreuth III: European consensus proposals for treatment of haemophilia with coagulation factor concentrates. Haemophilia. 2014 May;20(3):322-5. doi: 10.1111/hae.12440.
INTRODUCTION: This report summarizes recommendations relating to haemophilia therapy arising from discussions among experts from 36 European countries during the ‚Kreuth IV‘ meeting in May 2016. AIM: The objective of the meeting was for experts in the field of haemophilia from across Europe to draft resolutions regarding current issues relating to the treatment of haemophilia. RESULTS: Hospitals providing clinical care for people with haemophilia and related disorders are strongly recommended to seek formal designation as either European Haemophilia Treatment Centres (EHTC) or European Haemophilia Comprehensive Care Centres (EHCCC). There should be agreed national protocols or guidelines on management of the ageing patient with haemophilia. The minimum consumption of factor VIII and IX concentrate in any country should be 4 IU and 0.5 IU per capita of general population respectively. Treatment for hepatitis C with direct-acting antiviral agents should be provided to all people with haemophilia on a priority basis. Genotype analysis should be offered to all patients with severe haemophilia. Genetic counselling, when given, should encompass the recommendation that genetic relatives of the affected person be advised to seek genetic counselling. People with inhibitors should have access to bypassing agents, immune tolerance and elective surgery. National or regional tenders for factor concentrates are encouraged. Outcome data including health related quality of life should be collected. Treatment with extended half-life factors should be individualized and protection against bleeding should be improved by increasing trough levels. Steps should be taken to understand and minimize the risk of inhibitor development. CONCLUSION: It is hoped that these recommendations will help to foster equity of haemophilia care throughout Europe. Giangrande PLF et al.: Kreuth IV: European consensus proposals for treatment of haemophilia with coagulation factor concentrates. Haemophilia. 2017 May;23(3):370-375. doi: 10.1111/hae.13211. Epub 2017 Apr 12.