Autologous haematopoietic stem cell therapy for multiple sclerosis: a review for supportive care clinicians on behalf of the Autoimmune Diseases Working Party of the European Society for Blood and Marrow Transplantation

Abstract

Purpose of reviewIn this review, we summarize the recently published literature that demonstrates the efficacy and safety of autologous haematopoietic stem cell therapy (AHSCT) in multiple sclerosis (MS) and highlight the importance of supportive care required for the safe and well-tolerated delivery of AHSCT. Recent findingsMS is an autoimmune inflammatory and degenerative disorder of the central nervous system (CNS). In the majority of patients, the illness runs a relapsing remitting course (RRMS), culminating in a secondary progressive phase with gradual accumulation of fixed disabilities. Currently available disease-modifying therapies suppress CNS inflammation but have a limited effect on preventing disease progression for which there remains no effective therapy. Over the last two decades, there has been increasing evidence that AHSCT is a highly effective therapeutic strategy for treatment-resistant inflammatory types of MS, especially RRMS. Concerns about the safety of AHSCT in MS, usually a nonlife-threatening disease, have previously limited its use. However, AHSCT can now be delivered safely with major long-term benefits because of increasing transplant centre experience, judicious patient selection and good supportive care. SummaryMS is currently the fastest growing indication for AHSCT in Europe. Supportive care before, during and after the transplant period is key to the successful delivery of AHSCT.

Rehabilitation Before and After Autologous Haematopoietic Stem Cell Transplantation (AHSCT) for Patients With Multiple Sclerosis (MS): Consensus Guidelines and Recommendations for Best Clinical Practice on Behalf of the Autoimmune Diseases Working Party, Nurses Group, and Patient Advocacy Committee of the European Society for Blood and Marrow Transplantation (EBMT)

“…AHSCT provides a "one-off " treatment as opposed to DMTs, which generally require on-going administration (7,16). AHSCT, however is an intensive chemotherapy-based procedure that can result in deconditioning and which may add further to functional impairment, at least temporarily (17). AHSCT and its associated short-and long-term risks require counseling, detailed workup and an admission to specialized HSCT facilities (18).…”

“…For detailed general and MS specific literature on AHSCT, please refer to EBMT guidelines for AHSCT in MS and neurological diseases (14,17,28,29) and recent reviews, which cover the transplant process itself and its us in MS.…”

“…5. Post-transplant care Following stem cell infusion, the patient remains in hospital for a period of close monitoring, supportive care for side effects of treatment, and isolation from potential sources of infection while awaiting engraftment of the haematopoietic system, usually defined as the recovery of a neutrophil count of 10 9 /L for three days (17), which in most cases occurs 10-14 days following the infusion of the stem cells.…”

“…Potential early side effects include fever, infection, and sepsis in association with leukopenia, other cytopenias (anemia and thrombocytopenia) requiring blood product transfusions, and a range of other organ toxicities (alopecia, mucositis, diarrhea, vomiting, renal, and fluid balance issues and serum sickness). Worsening of existing neurological symptoms can occur often in the context of fever, drug reactions, and infection (17). Neurological deterioration in response to fever is known as the Uhthoff phenomenon, and, whilst the effect on patients during AHSCT is usually short lived and reversible, some evidence suggests the potential for prolonged deleterious effect on neurological function (32).…”

“…Secondary complications include deconditioning, reduced caloric intake due to nausea and mucositis and limited activity in the immediate post-transplant period (32). Prolonged bed rest and isolation during AHSCT may also lead to pressure sores, thromboembolic risks, changes in spasticity and posture, acquired urinary or bowel dysfunction, bone loss, vitamin D deficiency, low mood, and fatigue (17).…”