Members from four EBMT Working Parties and Committees have produced a position statement providing general information for patients and carers considering HSCT for severe autoimmune diseases.
General information for patients and carers considering haematopoietic stem cell transplantation (HSCT) for severe autoimmune diseases (ADs): A position statement from the EBMT Autoimmune Diseases Working Party (ADWP), the EBMT Nurses Group, the EBMT Patient, Family and Donor Committee and the Joint Accreditation Committee of ISCT and EBMT (JACIE)
Jessop H et al. Bone Marrow Transplant. 2019 Jan 31. doi: 10.1038/s41409-019-0430-7.
In the last 20 years the use of mainly autologous HSCT to treat severe autoimmune diseases such as relapsing remitting Multiple Sclerosis, Systemic Sclerosis/Scleroderma, Crohn’s disease and other diseases has increased dramatically, especially for patients whose response to conventional disease modifying treatments has been limited. The aim of this treatment is to ‘re-set’ or ‘re-boot’ the immune system, allowing for recovery of the damaged organ. This rapidly increasing use of HSCT treatment worldwide has prompted us (representatives of patients, carers, HSCT and disease-specialist clinicians and JACIE accreditation) to publish a set of plainly written open access guidelines to help inform the decision making process of both clinicians and patients. In this position paper, we provide a summary of the process of HSCT and have listed a set of recommendations to be considered by patients and their carers (whether they be family, friends or professional carers, such as their GPs or other non-specialist clinicians), based on the published literature and best practice.
We have emphasised the importance of close collaboration between disease specialists and experienced accredited transplant centres with careful consideration of the likely benefits of treatment, the immediate risks during the HSCT process, including treatment related mortality and the possibility of late onset treatment related complications (or ‘late effects’). This includes the need for close ongoing post transplant follow-up, irrespective of whether the patient has been treated close to their home base or abroad. EBMT member transplant centres with experience generally and in specific autoimmune diseases are referenced as potential sources of advice, support and/or referral for less experienced centres, where necessary.
Members from four EBMT working parties and committees have come together in this position statement, which we hope will provide patients, non-specialist clinicians and others supporting them with a clear representation of the HSCT process. We also hope that this information will be disseminated widely through patient groups internationally, and, ideally, translated to maximise worldwide access.
“I hope this EBMT position paper will provide clear non-judgemental guidance of the HSCT process and understanding of the balance of potential benefits and risks in order to support informed personalised decision making in this complex and evolving area of multidisciplinary practice.” Professor John Snowden, Chair of the EBMT Autoimmune Diseases Working Party and Chair of the JACIE Committee.
“The Patient Family and Donor Committee finds it very important to really bring the needs of patients and patients organisations to the fore. This position paper provides for this by providing concrete recommendations for patients with ADs considering a HSCT. ” Bregje Verhoeven, MSc, Chair of the EBMT Patient Family and Donor Committee.