Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Effective treatment for rare blood disorder

11.01.2019

Anyone who survives an acute episode of the rare blood disorder TTP is often left with long-term consequences, especially neurological damage. An international study, with the participation of the Department of Hematology of Bern University Hospital, was now able to verify the effectiveness of a new innovative treatment.

The life-threatening blood disorder thrombotic thrombocytopenic purpura (TTP) is rare (2-3 people out of every million per year) and affects mainly young, otherwise healthy people – women more frequently than men. TTP will lead to death if not treated within a few days.


Blood smear of a TTP patient: red blood cells are injured in the microcirculation, partially occluded by VWF-platelet clumps and compensatory reticulocytosis. Platelets are largely lacking

Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital

In healthy individuals, the protein scissor ADAMTS13 cleaves ultra large von Willebrand factor (VWF) multimers. Smaller VWF multimers are less sticky, do not bind platelets spontaneously and thus formation of blood clots in blood vessels is prevented.

In TTP, ADAMTS13 is lacking due to circulating autoantibodies. In the absence of VWF size regulation, platelets are consumed in VWF-platelet clots, that occlude the microcirculation. Blood circulation to end organs is decreased, resulting in heart attack, stroke and kidney failure. Hence TTP is also been refered to as “clumping plague”.

So far, standard treatment of acute episodes of TTP consists of a daily plasma exchange (to remove autoantibodies and replenish ADAMTS13) in combination with immunsuppressive drugs (to inhibit autoantibody formation).

Nevertheless, 10 - 20 percent of patients die during an acute episode. In addition, more than half of the survivors have permanent organ damage and dysfunction, particularly in the form of neurological deficits, and frequently experience relapses.

Rapid control of the disorder with lower rate of relapses

A major international study, with the participation of the Department of Hematology of Bern University Hospital, was published on January 9, 2019, in the New England Journal of Medicine (NEJM). The study was able to confirm the effectiveness of an innovative TTP treatment. The studied anti-VWF nanobody, caplacizumab, was shown to effectively prevent VWF-platelet clumping and thus protect the end organs from further depriviation of blood circulation.

145 TTP patients partcipated in this randomized Phase III study, 72 received the nanobody during the plasma exchange treatment and for 30 days thereafter, 73 received a placebo for the same period of time. In 75 percent of patients who received the study drug, the acute phase of TTP ended after 2.95 days, compared to 4.5 days with the conventional treatment.

Furthermore, the patients required fewer plasma exchange sessions (median 5 vs. 7), and could be discharged earlier. The side-effects were comparable in both study arms, although mild bleeding symptoms occurred more frequently with the new compound (65% compared to 48% with placebo). The study was able to confirm the promising data of the Phase II study, in which the Bern-based Hematology Department had also participated.

Long-term research topic at the Bern University Hospital and Bern University

The Department of Hematology at the Bern University Hospital and Bern University (Hematology Research Group, Department of BioMedical Research) have been researching TTP and ADAMTS13 since the mid-1990s. This resarch was initiated by Prof. em. Dr. phil nat. Miha Furlan and Prof. em. Dr. med. Bernhard Lämmle with the discovery of the von Willebrand factor-cleaving protease (now ADAMTS13), and the observation of its deficiency in TTP, and is currently headed by Prof. Dr. med. Johanna Kremer Hovinga. Since 2010 she has been involved in developing the new treatment approach for TTP, which will be available to all TTP patients in the future.

Wissenschaftliche Ansprechpartner:

Prof. Dr. med. Johanna A. Kremer Hovinga Strebel, Senior Consulting Physician, Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, Johanna.Kremer@insel.ch

Originalpublikation:

DOI: 10.1056/NEJMoa1806311

Weitere Informationen:

https://www.nejm.org/doi/full/10.1056/NEJMoa1806311

Monika Kugemann | idw - Informationsdienst Wissenschaft
Further information:
http://www.insel.ch

More articles from Life Sciences:

nachricht Rising water temperatures could endanger the mating of many fish species
03.07.2020 | Alfred-Wegener-Institut, Helmholtz-Zentrum für Polar- und Meeresforschung

nachricht Moss protein corrects genetic defects of other plants
03.07.2020 | Rheinische Friedrich-Wilhelms-Universität Bonn

All articles from Life Sciences >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Electrons in the fast lane

Solar cells based on perovskite compounds could soon make electricity generation from sunlight even more efficient and cheaper. The laboratory efficiency of these perovskite solar cells already exceeds that of the well-known silicon solar cells. An international team led by Stefan Weber from the Max Planck Institute for Polymer Research (MPI-P) in Mainz has found microscopic structures in perovskite crystals that can guide the charge transport in the solar cell. Clever alignment of these "electron highways" could make perovskite solar cells even more powerful.

Solar cells convert sunlight into electricity. During this process, the electrons of the material inside the cell absorb the energy of the light....

Im Focus: The lightest electromagnetic shielding material in the world

Empa researchers have succeeded in applying aerogels to microelectronics: Aerogels based on cellulose nanofibers can effectively shield electromagnetic radiation over a wide frequency range – and they are unrivalled in terms of weight.

Electric motors and electronic devices generate electromagnetic fields that sometimes have to be shielded in order not to affect neighboring electronic...

Im Focus: Gentle wall contact – the right scenario for a fusion power plant

Quasi-continuous power exhaust developed as a wall-friendly method on ASDEX Upgrade

A promising operating mode for the plasma of a future power plant has been developed at the ASDEX Upgrade fusion device at Max Planck Institute for Plasma...

Im Focus: ILA Goes Digital – Automation & Production Technology for Adaptable Aircraft Production

Live event – July 1, 2020 - 11:00 to 11:45 (CET)
"Automation in Aerospace Industry @ Fraunhofer IFAM"

The Fraunhofer Institute for Manufacturing Technology and Advanced Materials IFAM l Stade is presenting its forward-looking R&D portfolio for the first time at...

Im Focus: AI monitoring of laser welding processes - X-ray vision and eavesdropping ensure quality

With an X-ray experiment at the European Synchrotron ESRF in Grenoble (France), Empa researchers were able to demonstrate how well their real-time acoustic monitoring of laser weld seams works. With almost 90 percent reliability, they detected the formation of unwanted pores that impair the quality of weld seams. Thanks to a special evaluation method based on artificial intelligence (AI), the detection process is completed in just 70 milliseconds.

Laser welding is a process suitable for joining metals and thermoplastics. It has become particularly well established in highly automated production, for...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

VideoLinks
Industry & Economy
Event News

International conference QuApps shows status quo of quantum technology

02.07.2020 | Event News

Dresden Nexus Conference 2020: Same Time, Virtual Format, Registration Opened

19.05.2020 | Event News

Aachen Machine Tool Colloquium AWK'21 will take place on June 10 and 11, 2021

07.04.2020 | Event News

 
Latest News

Rising water temperatures could endanger the mating of many fish species

03.07.2020 | Life Sciences

Risk of infection with COVID-19 from singing: First results of aerosol study with the Bavarian Radio Chorus

03.07.2020 | Studies and Analyses

Efficient, Economical and Aesthetic: Researchers Build Electrodes from Leaves

03.07.2020 | Power and Electrical Engineering

VideoLinks
Science & Research
Overview of more VideoLinks >>>