Haematopoietic stem cell transplants: principles and indications

Abstract

Haematopoietic stem cell transplantation was proposed as a treatment strategy just over 60 years ago. Owing to great advances in the field, haematopoietic stem cell transplantation has become an established method for the treatment of many haemato-oncological, immunological and hereditary conditions with the potential of cure. The number of haematopoietic stem cell transplants performed worldwide reached one million by 2012. This review provides an overview of autologous and allogeneic haematopoietic stem cell transplantation including disease indications, the individual steps of the procedure and outcome, and highlights achievements in the treatment of autoimmune diseases. Although autoimmune conditions account for only 1% of indications for autologous haematopoietic stem cell transplant, this is increasingly used to treat high-risk autoimmune diseases. Haematopoietic stem cell transplantation can induce long-term remission by resetting the immune system via eradication of autoreactive immune cells and the generation of a de novo self-tolerant immune system. Data seem most encouraging in multiple sclerosis and systemic sclerosis and it is likely that the number of procedures performed to treat these conditions will rise in the future.

A Review of Infections After Hematopoietic Cell Transplantation Requiring PICU Care: Transplant Timeline Is Key

“…The goal of a HCT is to eliminate a patient's native bone marrow (BM) cells and to replace them with healthy donor cells ( 11 ). These native stem cells may contain a genetic mutation that is driving a certain disease state, or may be defective resulting in BM failure, or a neoplasm that cannot be destroyed with conventional chemotherapy or radiation ( 11 ).…”

“…The goal of a HCT is to eliminate a patient's native bone marrow (BM) cells and to replace them with healthy donor cells ( 11 ). These native stem cells may contain a genetic mutation that is driving a certain disease state, or may be defective resulting in BM failure, or a neoplasm that cannot be destroyed with conventional chemotherapy or radiation ( 11 ). To destroy these errant stem cells, high dose chemotherapy and often radiation, are administered to ablate the BM to make space for new stem cells to begin the process of renewed hematopoiesis ( 12 ).…”

“…It is important to understand that the very nature of an allogenic transplant necessitates the use of immunosuppression for a short time to allow for the immunologic establishment of the new stem cells ( 17 ). For some conditions, the cure can be achieved with high dose chemotherapy followed by the infusion of a patient's own, previously collected, stem cells ( 11 ). This is termed an autologous HCT ( 11 ).…”

Zukunftsweisende Therapieansätze bei Riechstörungen: elektrische Stimulation, Stammzelltherapie und Transplantation von Riechepithel – eine Übersicht

“…Die erste erfolgreiche Nierentransplantation erfolgte in Paris schon im Jahr 1953 [27]. Am Beispiel von hämatoonkologischen Patienten lässt sich zudem zeigen, wie groß der Stellenwert von Stammzelltransplantationen in unseren heutigen Therapiekonzepten ist [4]…”

Successful use of allogeneic bone marrow transplantation in a patient with myelodysplastic syndrome presenting with autoimmune manifestations

“…It is in this group of patients that HSCT represents an alternative therapeutic strategy, which may offer sustained remission by more potent eradication of auto‐reactive T cells and development of self‐tolerance 9 . Overall, only 0·1% of allo‐BMTs are performed for primarily autoimmune disease 10 . There are limited data on the incidence of allogeneic transplantation performed for autoimmune disease associated with MDS, but case reports are described 11–12 …”