[Dr.Vad]

специально пройдусь по применению АТКМ при аутоимунных заболеваниях по отдельным нозологиям:
(Popat U, Krance R. Haematopoietic stem cell transplantation for autoimmune disorders: the American perspective.Br J Haematol. 2004 Sep;126(5):637-49.)

Multiple sclerosis

Haematopoietic stem cell transplantation is a feasible option with a low (<5%) mortality rate in patients with MS. Worsening of neurological function with exclusive use of G-CSF for mobilization, an increased frequency of engraftment syndrome, and transient worsening of neurological deficits with fever in the post-transplant phase are unique complications of this procedure in patients with MS. The best and least toxic conditioning regimen remains to be determined. Although patients with advanced disability are less likely to benefit from HSCT, this procedure remains a promising approach, particularly for patients in the early stages of disease and with an EDSS < 6-6,5.

Systemic sclerosis

Autologous HSCT appears feasible in patients with Ssc. High treatment-related mortality rates seen in the initial trials can be reduced with better patient selection and protocol modifications. Improvements in skin scores and quality of life are very promising and have not been achieved previously with any other therapy. Phase III studies currently underway in Europe and being planned in the USA should clarify the impact of autologous HSCT on the natural history of Ssc.

Systemic lupus erythematosus

Haematopoietic stem cell transplantation offers a chance for disease control and an improved quality of life to patients with refractory SLE. The reported mortality associated with this procedure reflects the advanced organ dysfunction of most transplanted patients. More experience and better patient selection will undoubtedly reduce treatment-related mortality to an acceptable level. Currently planned phase III trials should clarify the place of autologous HSCT in the therapeutic armamentarium for SLE.

Rheumatoid arthritis

It is evident from these studies that HSCT has minimal toxicity in RA patients. Two-thirds of the patients respond to transplantation, but the responses are not sustained and the majority of patients relapse. However, a substantial proportion of relapsed patients respond to DMARD, many after showing resistance to the same agents in previous treatment. Maintenance therapy post-transplantation, intensification of the conditioning regimen or allogeneic transplantation are all possible options for improving clinical outcome. Most previously described patients with RA were treated before the availability of TNF-blocking agents and other biological response modifiers. With the availability of these safe and effective agents, the number of patients with refractory disease suitable for HSCT trials has decreased. Randomized trials currently under consideration for patients failing TNF-blocking agents may clarify the role of HSCT in the management of severe RA.

Haematological disorders

Of the 12 patients with refractory ITP who underwent autologous transplantation and were reported to the EBMT registry, two died of toxicity while four had a durable response. In addition to patients with ITP, this study also included patients who had an autologous or allogeneic transplant for Evan's syndrome, autoimmune haemolytic anaemia, thrombotic thrombocytopenic purpura and pure white cell aplasia. Continuous response was seen in nine of 26 patients having an autograft and five of nine patients undergoing an allograft, but four of 34 patients died from treatment-related toxicity. Further studies are needed to define the role of HSCT in patients with refractory cytopenias.

Juvenile idiopathic arthritis

Juvenile idiopathic arthritis is another disease for which HSCT may be useful. In an early report, four children with resistant disease attained complete remission. Since then, more than 40 children have been treated, with three dying of macrophage activation syndrome, also called infection-associated haemophagocytic syndrome. The majority of patients treated in this manner responded.

Other autoimmune diseases

Haematopoietic stem cell transplantation has been studied in other autoimmune diseases that are refractory to standard therapy. Four patients with refractory Crohn's disease were treated on a protocol in which CD34-selected autologous peripheral blood stem cells were infused after conditioning with cyclophosphamide and ATG. All four patients achieved remission after relapsing on multiple standard therapies. Further follow-up is needed to determine the durability of these remissions.

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Autologous HSCT has been studied in patients with refractory autoimmune diseases since the mid-1990s. Early studies have produced mixed results, with marked improvements in skin involvement and quality of life observed in patients with SLE and Ssc. Stabilization of disease has been documented in two-thirds of patients with MS; however, for patients with RA, significant initial benefits have been followed by eventual relapse. With growing clinical experience and modification of the protocols, the treatment-related mortality rate is decreasing. Wider application of HSCT in autoimmune diseases will require proof of efficacy in ongoing randomized phase III trials comparing transplantation with standard therapy.

PS. Подробнее о месте трансплантации КМ именно при лечении аутоиммунных, незлокачественных гематологических заболеваний и злокачественных опухолей можно почерпнуть абсолютно бесплатно в ежегодном обучающем манускрипте от ASH 2003 г. Hematopoietic Cell Transplantation for Benign Hematological Disorders and Solid Tumors:

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