Nonmyeloablative allogeneic bone marrow transplantation for treatment of childhood overlap syndrome and small vessel vasculitis

Abstract

Summary:A 13-year-old Caucasian female with a systemic connective tissue disease (overlap syndrome with pulmonary vasculitis) underwent nonmyeloablative allogeneic BMT after failure of prolonged combination immunosuppressives to induce remission. The procedure also included cotransplantation of donor bone chips as a source of stromal cells. The unique protocol allowed good engraftment of hematopoietic (495%) and bone core stromal cells (460%). The patient was clinically improved, stable, and off all immunosuppressive medications 36 months post-transplant. To our knowledge, this is the first pediatric nonmyeloablative BMT with cotransplantation of stromal cells solely for treatment of an autoimmune disease.

Nonmyeloablative allogeneic bone marrow transplantation of a child with systemic autoimmune disease and lung vasculitis

“…Her diffusion capacity (DLCO) was 65% predicted that remained at 60% after the first year of aggressive treatment with monthly IV cyclophosphamide and high doses of steroids. Her CT findings resolved and she did not [1] have evidence of pulmonary hypertension by echocardiogram and cardiac catheterization. Clinically she was cushinoid, had symptoms of fatigue, intermittent periods of mild rash on the hands (gottron's-like), knees and elbows, as well as proximal muscle weakness correlating with a mild increase in aldolase and von Willebrand factor antigen.…”

“…Here, we present a description of the last transplant case that he mentored in the year 2000. This case represents the first successful nonmyeloablative bone marrow transplant of a child providing long-term remission of her systemic autoimmune disease and small vessel vasculitis [1].…”

Reduced-intensity allogeneic hematopoietic stem cell transplantation for acute leukemias: ‘what is the best recipe?’

“…90,91 Future studies may also attempt to 'target' the kinetics of complete donor T-cell chimerism according to the need for immediate disease control. The indications for RIST may therefore expand beyond those of MAST.…”

Transplante de células-tronco hematopoéticas em doenças reumáticas parte 1: experiência internacional

“…Curiosamente, em uma paciente que recebeu TCTH autólogo para poliarterite nodosa juvenil em Londres, Reino Unido, apesar de a doença ter entrado em remissão após 18 meses de seguimento, surgiram outras DAIs após o transplante (vasculite cutânea, doença de Graves e púrpura trombocitopênica auto-imune), que foram controladas com tratamento adequado (72) . Por outro lado, uma paciente de 13 anos de idade, portadora de uma síndrome de superposição auto-imune, com vasculite pulmonar, entrou em remissão com um transplante alogênico não-mieloablativo combinado de medula óssea, espículas ósseas e osteoblastos cultivados in vitro, após um condicionamento também subablativo (73) .…”