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05.07.2016

Haematopoietic stem cell transplantation in the Netherlands for severe generalized RDEB

Patients with severe recessive dystrophic EB (RDEB) have a life expectancy of an average 35 years and eventually die of skin cancer in the wounds. The disease is not curable.

A new therapy that may improve (but not cure) the condition is allogeneic haematopoietic stem cell transplantation (HSCT). HSCT has a survival rate of 80-85%. HSCT for EB is also available in the USA. Over the past year we have adjusted our treatment protocol to optimalize safety and reduce the risk of mortality occurring from treatment.

In the Netherlands, allogeneic HSCT using cord blood from an unrelated donor is covered by health insurance. Due to the novelty, the treatment is being offered in the form of a clinical trial.

The trial has been open as of March 1st 2016 in the University Medical Centre Groningen and Wilhelmina Children’s Hospital of the University Medical Centre Utrecht. The rate for intake and transplantation is limited to one patient per two months. We aim to treat 10 patients.

Eligible patient are those with severe generalized recessive dystrophic epidermolysis bullosa (preferably complete absence of type VII collagen), an age between 0-18 years, and be willing to visit Groningen and Utrecht regularly for 2 years. The hospitalization period is on average 8 weeks but may be longer.

Patients from the European Union need an E-112 form approved by their national health insurance to cover hospital costs in the Netherlands. Parents can rent a room in the Ronald McDonald House or find a rental apartment with our assistance. Parents have to cover their own costs for travel and stay in the Netherlands for the duration of treatment and follow-up.

Severe generalized junctional epidermolysis bullosa (Herlitz) is not an indication that is eligible for this research trial. 

For all inquiries, please contact:

Katarzyna Gostynska, M.D. PhD, Coordinating trial investigator k.gostynska(at)umcg.nl

Centre for Blistering Diseases, Department of Dermatology

University Medical Centre Groningen
Hanzeplein 1, Groningen, the Netherlands

Authors:

Katarzyna Gostynska, M.D. PhD

Prof. dr. M.F. Jonkman, dermatologist Centre for Blistering Diseases Department of Dermatology University Medical Centre Groningen 

Dr. J.J. Boelens, pediatric oncologist-immunologist Pediatric Bone Marrow and Blood Transplantation Program Wilhelmina Children’s Hospital University Medical Centre Utrecht