Teng, Y. K. et al. Long-term followup of health status in patients with severe rheumatoid arthritis after high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation. Arthritis Rheum. 52, 2272-2276

Leiden University, Leyden, South Holland, Netherlands
Arthritis & Rheumatology (Impact Factor: 7.76). 08/2005; 52(8):2272-6. DOI: 10.1002/art.21219
Source: PubMed


High-dose chemotherapy (HDC) followed by autologous hematopoietic stem cell transplantation (HSCT) is a new treatment for patients with severe, refractory rheumatoid arthritis (RA). The present study was undertaken to assess the health status of patients with severe RA over a long-term followup period after treatment with HDC + HSCT.
Health status and utility scores were assessed in 8 patients before and after treatment with HDC + HSCT. Patients were followed up for 5 years posttransplantation. Health status was assessed by the Health Assessment Questionnaire (HAQ), the RAND-36 version of the Short Form 36 (SF-36) health survey, and the Arthritis Impact Measurement Scales (AIMS). Utility scores were calculated using the EuroQol (EQ-5D) questionnaire and the SF-36-derived utility index (called the SF-6D), from which quality-adjusted life years (QALYs) were derived.
Most measures of health status improved compared with baseline in the first 2 years posttransplantation, notably HAQ and AIMS scores and scores on the functional status, general health, and health change summary scales of the RAND-36 version of the SF-36. Utility scores derived from the EQ-5D questionnaire and the SF-6D also increased significantly after transplantation. This was reflected in the 0.28 QALYs gained compared with baseline. For a putative 50-year-old RA patient with a life expectancy of 20 years, a threshold analysis revealed that HDC + HSCT yielded more QALYs than conventional therapy when treatment-related mortality (TRM) was <2.8%.
HDC + HSCT temporarily increased the functionality and health status of patients with severe, refractory RA. With a reported TRM of 1.3%, HDC + HSCT can be considered a realistic treatment option for patients with severe RA.

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Available from: Y K O Teng, Oct 16, 2014
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    • "Although the approach to use high dose myeloablative therapy combined with subsequent hematopoietic stem cell transplantation (HSCT) was first described more than 50 years ago for the treatment of malignant conditions, this principle was adopted in recent years for treatment of various autoimmune diseases. It is evident that complete immunoablation is a drastic way to achieve maximal treatment efficiency in autoimmune diseases (Teng et al., 2005), with potentially lethal complications such as cardiotoxicity or overt opportunistic infections. For this, HSCT is only considered in patients suffering from severe and progressive autoimmune disease and refractory to conventional immunosuppressants. "

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    • "In SLE patients, disease activity as measured by the Systemic Lupus Erythematosus Disease Activity Index improved markedly [26,27]; and in those patients with pulmonary abnormalities, lung function tests showed significant improvements in the years following HSCT [28]. In contrast, most RA patients showed only transient responses, as measured by scores of disease activity, functional ability, quality of life, and rate of joint destruction, although the disease appeared more amenable to antirheumatic medication post HSCT [29,30]. "
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    ABSTRACT: Cell therapy, pioneered for the treatment of malignancies in the form of bone marrow transplantation, has subsequently been tested and successfully employed in autoimmune diseases. Autologous haemopoietic stem cell transplantation (HSCT) has become a curative option for conditions with very poor prognosis such as severe forms of scleroderma, multiple sclerosis, and lupus, in which targeted therapies have little or no effect. The refinement of the conditioning regimens has virtually eliminated transplant-related mortality, thus making HSCT a relatively safe choice. Although HSCT remains a nonspecific approach, the knowledge gained in this field has led to the identification of new avenues. In fact, it has become evident that the therapeutic efficacy of HSCT cannot merely be the consequence of a high-dose immuno-suppression, but rather the result of a resetting of the abnormal immune regulation underlying autoimmune conditions. The identification of professional and nonprofessional immunosuppressive cells and their biological properties is generating a huge interest for their clinical exploitation. Regulatory T cells, found abnormal in several autoimmune diseases, have been proposed as central to achieve long-term remissions. Mesenchymal stem cells of bone marrow origin have more recently been shown not only to be able to differentiate into multiple tissues, but also to exert a potent antiproliferative effect that results in the inhibition of immune responses and prolonged survival of haemopoietic stem cells. All of these potential resources clearly need to be investigated at the preclinical level but support a great deal of enthusiasm for cell therapy of autoimmune diseases.
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