From Clinica Ruiz
Hematopoietic stem cell transfusion for multiple sclerosis Clinica Ruiz
Multiple sclerosis (MS) is a chronic inflammatory and / or axonal degenerative disease that causes destruction of central nervous system (CNS) myelin and nervous tissue pathways, with varying degrees of axonal damage. It predominantly affects young adults (the symptoms appear around 30 years of age, on average), and is twice as common in women as in men. Studies published from the 1990s brought animal models and theoretical considerations of hematopoietic stem cell transplantation (HSCT) in the prevention and treatment of autoimmune diseases with a favorable clinical response in many patients, suggesting that high-dose chemotherapy followed by hematopoietic stem cell rescue could ?reset? the pathological immunological dysfunction through the control of autoreactive lymphocytes and lymphocytic clones, followed by restoration of immunological tolerance after immune reconstitution. This led to the conclusion that HSCT may be a viable therapeutic option for MS, and other hematologically-rooted autoimmune diseases. Autologous HSCT has been done in patients with MS since the mid-1990?s, and several thousands of HSCTs have been performed around the world for MS since, with patients transplanted in clinical trials, multicenter studies, and private treatment. In retrospective analyses, a progression-free survival of more than five years after transplant has been observed for the majority of recipients, with post-transplantation neurological outcomes being more favorable in patients with the episodic (relapsing-remitting) type, and/or those who showed an inflammatory pattern in magnetic resonance imaging (MRI) during the pre-transplant screening. Reports of good results, particularly in the aggressive forms of MS, reinforce the effectiveness HSCT in MS patients with prominent inflammatory activity, and a halting of underlying disease progression in the absence of inflammatory activity for the progressive form of the disease. The risk of transplant related mortality in HSCT for MS was previously conventionally considered high, but has significantly declined since 2001 to currently less than 1% according to the European Group for Blood and Marrow Transplantation (EBMT, www.ebmt.org) analysis, this probably being the result of the changes in the contemporary conditioning regimens and proactive patient monitoring, thus reducing toxicity, morbidity and mortality. The consensus on HSCT also provides an indication of HSCT in patients with progressive MS unresponsive to conventional therapy. The forms of the disease that might benefit from transplantation are: relapsing remitting, primary or secondary progressive, relapsing-progressive, and the ?malignant? forms of MS (Tumefactive, Balo, Marburg variant).
Since 1993, scientists from the Centro de Hematolog?a y Medicina Interna de Puebla (CHMI) have engaged in practicing HSCT using novel methods to both decrease the toxicity of the procedures and diminish costs. Over 400 HSCTs have been done in the CHMI for different diseases such as acute leukemia, chronic leukemia, aplastic anemia, myeloma, lymphomas, myelodysplasias and autoimmune diseases, including MS.
- Patients with MS having a confirmed positive diagnosis including Relapsing ? Remitting (RRMS), Secondary Progressive (SPMS), Primary Progressive (PPMS) and Progressive Relapsing (PRMS).
- Patients must have a Karnofsky performance status above 70% and an EDSS score of 7 or below.
- All patients accepted for treatment at CHMI must have a properly capable and sufficiently competent carer accompanying the patient.
- A recent central nervous system (CNS) MRI study (less than three months) must be provided.
- Patient has to be able to travel to, and remain in Puebla, M?xico during a 4-week period, accompanied by a caregiver.