Hematopoietic Stem Cell Transplantation for the Treatment of Severe Autoimmune Diseases

Tyndall, Alan; Harmatz, Paul

doi:10.1007/0-387-24534-0_24

Cited by 3 publications

(5 citation statements)

References 51 publications

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“…In our study, rapid evolution towards death or end‐stage organ failure in the six eligible patients who delayed the procedure, reflected the severity of the disease. On the other hand, partial or major response observed in two‐thirds of those who underwent autologous PBSC or bone marrow transplantation showed that intensification and autologous transplantation can improve the short‐term survival in severe SSc, as suggested previously by heteregeneous data reported to the EBMT‐EULAR (European League Against Rheumatism (EULAR) and the European Group for Blood and Marrow Transplantation (EBMT)) registry (Tyndall et al, 2001). Immunosuppression is the best current therapeutic option in diffuse severe SS and the rationale for high‐dose chemotherapy and autologous PBSC or BM transplantation in ADs has been extensively reviewed (Ikehara et al , 1985; Marmont, 1994, 1995; Tyndall & Gratwohl, 1997; Van Bekkum, 1998; Sherer & Shoenfeld, 1998; Farge et al , 1999; Gratwohl & Tyndall, 1999).…”

Section: Discussionsupporting

confidence: 59%

“…Because of the high mortality associated with allogeneic BMT (at least 30%, even without underlying malignancy), autologous peripheral blood stem cells (PBSC) or BM transplantation, with its low risk of early mortality (< 3–5%), was preferred for preliminary studies in ADs (Marmont et al , 1995; Tyndall & Gratwohl, 1997). Approximately 400 autologous BM or PBSC transplantations were reported world‐wide for various ADs in 2001 (Tyndall et al , 2001), but great heterogeneity in the indications and the local practices required homogeneous clinical studies. We therefore designed the first multicentre, sequential phase I–II study to determine the feasibility of PBSC mobilization and positive CD34 + selection to achieve T‐cell depletion, the haematopoietic reconstitution after CD34 + reinfusion, its toxicity, and its potential efficacy in patients with refractory diffuse SSc.…”

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confidence: 99%

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Autologous bone marrow transplantation in the treatment of refractory systemic sclerosis: early results from a French multicentre phase I–II study

Farge¹,

Marolleau²,

Zohar

et al. 2002

Br J Haematol

126

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Summary. Haematopoietic stem cell transplantation (HSCT) has been proposed for refractory autoimmune diseases, including systemic sclerosis (SSc). A sequential Bayesian phase I-II clinical trial was conducted in SSc patients to assess the feasibility, the tolerance and the efficacy of autologous HSCT. Peripheral blood stem cells (PBSC) were collected using cyclophosphamide (4 g/m 2 ) and recombinant human granulocyte colony-stimulating factor (5 lg/kg/d) and reinfused after positive CD34 + selection. Conditioning used cyclophosphamide (200 mg/kg) or melphalan (140 mg/m 2 ) according to cardiac function. The main end-point was the failure of the procedure, defined by failure of either PBSC mobilization, CD34 + selection or intensification procedure, or by procedure-related death. Among the 12 enrolled patients, three failures occurred: one PBSC mobilization, one CD34 + selection and one CD34 + intensification. Probability of graft failure was estimated at 0AE286 (95% confidence interval: 0AE095-0AE54). Autologous PBSC (n ¼ 10) or bone marrow (n ¼ 1) transplantation was actually performed in 11 patients with one procedurerelated death. Median time to neutrophil (> 0AE5 · 10 9 /l) and platelet (> 25 · 10 9 /l) haematopoietic reconstitution was 12 and 10 d respectively. After 18 months (range 1-26), eight out of 11 patients have shown major or partial response. Non-myeloablative conditioning, followed by a T cell-depleted autologous PBSC or bone marrow transplantation, appears feasible with low toxicity in severe SSc with short-term clinical benefits.

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Section: Discussionsupporting

confidence: 59%

mentioning

confidence: 99%

Autologous bone marrow transplantation in the treatment of refractory systemic sclerosis: early results from a French multicentre phase I–II study

Farge¹,

Marolleau²,

Zohar

et al. 2002

Br J Haematol

126

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“…After collection of progenitor cells, most but not all centers carry out ex vivo lymphocyte depletion (31). Because the existence or identity of suppressor cells remains vague, graft depletion techniques might be non‐specific.…”

Section: Autologous Transplantation Of Cd34+ Cell‐selected Cells For mentioning

confidence: 99%

“…An excellent review on the immunology of transplantation tolerance underlying this novel therapeutic trial has been published recently (30). Data on 390 patients presented by Tyndall et al (31) included 127 cases of multiple sclerosis (MS), 72 cases of systemic sclerosis (SSc), 70 cases of rheumatoid arthritis (RA), 36 cases of juvenile idiopathic arthritis (JIA), 34 cases of systemic lupus erythematosus (SLE), five cases dermatomyositis/polymyositis (DM/PM), and idiopathic thrombocytopenic purpura (ITP), as the major disease categories. Clinically significant responses were seen in two‐thirds of all the cases and in all disease categories, with a more accentuated trend towards relapse in JIA and RA.…”

Section: Autologous Transplantation Of Cd34+ Cell‐selected Cells For mentioning

confidence: 99%

Clinical Applications of CD34+ Cell‐selected Peripheral Blood Stem Cells

et al. 2003

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Peripheral blood stem cells (PBSC) are increasingly used for stem cell transplantation after high dose chemotherapy. CD34+ cell selection has also been done for use in autologous transplantation studies Bone marrow (BM) may contain tumor cells at the time of harvesting, and on re-infusion, these cells could contribute to a subsequent relapse. Similarly, tumor cell contamination of PBSC collections has been found in a number of studies. Therefore, purging contaminating tumor cells may prevent cases of relapse. As most tumor cell types do not express CD34 antigen, one of the most widespread applications of CD34+ cell selection is likely to be in tumor cell purging. Similarly, CD34+ cell selection has aided allogeneic transplantation studies. Acute graft-versus-host disease (aGVHD) is a major cause of morbidity and mortality in cases of allogeneic transplantation. As aGVHD is mediated by donor T cells, removal of T cells from the graft by CD34+ cell selection may ensure prophylaxis against aGVHD. Further, high-dose immunosuppression followed by CD34+ cell-selected stem cell rescue is theoretically reasonable as a therapeutic tool for patients with autoimmune disease resistant to conventional therapy. However, patients given T cell-depleted transplantation have an increased risk of opportunistic infection as well as malignancies related to immunosuppression; therefore, close monitoring is warranted. We describe here clinical applications of CD34+ cell-selected PBSC for a variety of diseases, with special emphasis on the efficacy as well as drawbacks of this novel technique.

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“…Introdução A aplicação do transplante de células tronco hematopoéticas (TCTH) no tratamento de doenças auto-imunes (DAI) graves e refratárias possui sólidas bases experimentais e clínicas (1)(2)(3). Desde a década de 70, o sucesso desta abordagem terapêutica foi amplamente demonstrado em modelos animais de DAI e em pacientes portadores dessas doenças que foram transplantados para hemopatias concomitantes.…”

Section: Artigo Especialunclassified

Transplante de células tronco hematopoéticas para doenças auto-imunes no Brasil

Voltarelli¹

2002

Rev. Bras. Hematol. Hemoter.

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O transplante de células progenitoras hematopoéticas apresenta bases sólidas para ser indicado no tratamento das doenças autoimunes. No relato são apresentados estes dados e a seqüência de eventos que tem contribuído para a implantação de um protocolo brasileiro do procedimento na modalidade autogênica utilizando regimes de mobilização uniforme com Ciclofosfamida 2g/m 2 mais fator estimulador de colônias granulocitárias e condicionamentos particulares para o lúpus eritematoso sistêmico, esclerose sistêmica e esclerose múltipla. As entidades descritas anteriormente foram escolhidas para o início do protocolo cooperativo após amplos debates em encontros ocorridos em Ribeirão Preto e São .rancisco, EUA.

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Hematopoietic Stem Cell Transplantation for the Treatment of Severe Autoimmune Diseases

Cited by 3 publications

References 51 publications

Autologous bone marrow transplantation in the treatment of refractory systemic sclerosis: early results from a French multicentre phase I–II study

Autologous bone marrow transplantation in the treatment of refractory systemic sclerosis: early results from a French multicentre phase I–II study

Clinical Applications of CD34+ Cell‐selected Peripheral Blood Stem Cells

Transplante de células tronco hematopoéticas para doenças auto-imunes no Brasil

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