RT Journal Article
SR Electronic
T1 Autologous haematopoietic stem cell transplantation (aHSCT) for severe resistant autoimmune and inflammatory diseases – a guide for the generalist
JF Clinical Medicine
JO Clin Med
FD Royal College of Physicians
SP 329
OP 334
DO 10.7861/clinmedicine.18-4-329
VO 18
IS 4
A1 John A Snowden
A1 Basil Sharrack
A1 Mohammed Akil
A1 David G Kiely
A1 Alan Lobo
A1 Majid Kazmi
A1 Paolo A Muraro
A1 James O Lindsay
YR 2018
UL http://www.rcpjournals.org/content/18/4/329.abstract
AB Autologous haematopoietic stem cell transplantation (aHSCT) is commonly used for the treatment of haematological cancers, but is increasingly used in the treatment of patients severely affected by autoimmune diseases (ADs). In fact, ADs have become the fastest growing indication for aHSCT. A wide range of diseases have been treated, but the field has focused on three areas: multiple sclerosis, diffuse cutaneous systemic sclerosis and Crohn's disease, where there are populations of patients for whom disease control remains unsatisfactory despite the advent of biological and targeted small molecule therapies. Scientific studies of immune reconstitution have provided support for a ‘rebooting’ of the immune system through a re-diversification of naive and regulatory immune effector cells. In addition, there may be health economic benefits from a single one-off procedure. Even so, the treatment with aHSCT is intensive with a range of toxicities and risks which, despite being routine to transplant haematologists, are less familiar to disease specialists. Close collaboration between transplant haematologists and relevant disease specialists in patient selection, clinical management and follow-up is mandatory. Ideally, patients should be treated on a clinical trial if available.