Nonmyeloablative autologous hematopoietic stem cell transplantation for refractory CIDP
Department of Medicine, Division of Immunotherapy, Feinberg School of Medicine, Northwestern University, 750 N. Lakeshore Dr. 649, Chicago, IL 60611, USA. Neurology
(Impact Factor: 8.29).
11/2007; 69(18):1802-3. DOI: 10.1212/01.wnl.0000277266.53449.8b
Available from: Abraham C.J. Stork
- "Hematological stem cell therapy (SCT) is a well-established therapy used for refractory hematological malignancies. SCT has been reported to attenuate the progressive disease course of patients with polyneuropathy with organomegaly, endocrinopathy, M-protein and skin changes (POEMS) [5, 6], chronic inflammatory demyelinating polyneuropathy (CIDP) [7, 8], primary AL amyloidosis , and recently in a small series of patients with IgG MGUS- or MM-associated neuropathy , but it is unknown whether SCT may represent a rescue therapy for a wider range of patients with polyneuropathy and B cell dyscrasias and whether the possible beneficial effects outweigh risks associated with SCT. "
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ABSTRACT: B cell dyscrasias are often refractory to medical treatments, and hematological stem cell therapy (SCT) may be warranted. It is not clear whether an associated polyneuropathy may also profit from SCT. In exceptional cases SCT has been tried in patients with monoclonal gammopathy and progressive polyneuropathy refractory to medical treatments. In a cohort of 225 patients with monoclonal gammopathy and polyneuropathy, we selected the six patients who underwent SCT and retrospectively examined the effects of SCT on the disease course of the associated polyneuropathy. In all patients except one, the indication for SCT was hemato-oncological (multiple myeloma in 4 patients and primary AL amyloidosis in 1). The remaining patient had an IgG monoclonal gammopathy of undetermined significance and a progressive and painful polyneuropathy for which she was treated with SCT. SCT led to improvement of motor scores and autonomic symptoms in one patient; three patients experienced improvement of neuropathic pain or sensory deficits but showed further progression of weakness. One patient showed no improvement at all. One patient died within 100 days after SCT. In conclusion, SCT as a treatment of refractory hematological malignancy may occasionally have a positive effect on the associated progressive polyneuropathy, although the benefits are very limited and the treatment-related mortality is high.
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- "Last but not least, the costs of this treatment are approximately 30–50% lower. Since the first report in 2002, successful treatment of refractory CIDP patients with autologous stem cell transplantation has been reported (Oyama et al., 2007; Remé nyi et al., 2007; Axelson et al., 2008). In one small case series, 1/2 patients had transient improvement after this procedure (Mahdi- Rogers et al., 2007). "
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ABSTRACT: Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP)-related research has made progress in the field of pathogenesis, genetics, and treatment. The number of circulating CD4(+) CD25(+) T-regulatory cells was shown to be reduced in CIDP patients. Increased frequency of genotype GA13-16 of the SH2D2A gene encoding for a T-cell-specific adapter protein in CIDP patients may result in a defective control and elimination of autoreactive T cells. IVIg treatment has been shown to increase numbers and function of peripheral CD4(+) CD25(+) T-regulatory cell in a mouse model. These findings shed new light on the understanding of why peripheral tolerance is breached in CIDP patients and why the disease becomes chronic and adds another possible mechanism of action of intravenous immunoglobulin to the already long list. Long-term effectiveness of IVIg has now been proven. Subcutaneous immunoglobulin could be an alternative for IVIg, but this has to be explored further in well-designed trials. Autologous stem cell transplantation has been tried in refractory patients, but larger trials are necessary to assess safety and effect of this treatment.
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