Hematopoietic Stem Cell Transplantation in Chronic Inflammatory Demyelinating Polyneuropathy

Inclusion criteria

• Definite CIDP according to the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) criteria

AND

• Clinically typical or atypical CIDP

AND

• Failure to tolerate or respond to, or an incomplete response to, or relapse after at least 3 months of conventional treatment consisting of corticosteroids (equivalent dosage of prednisone 1.0/mg/day to 0.75mg/kg/day to start with adequate tapering trials of no less than 0.5mg/kg/day), and/or either intravenous immunoglobulin (IVIg) or plasmapheresis or cytoxan or rituxan
• Failure to respond to therapy is defined by:
• Persistent muscle weakness Grade 3/5 or worse (MRC) in at least one muscle or grade 4/5 in at least two muscle groups OR
• Persistent dysphagia documented by either aspiration or insufficient clearing on videofluoroscopic examination.

OR

• Persistent incapacitating sensory loss (e.g. gait ataxia, falls > 1/month)

AND

• If patients are on IVIG or plasmapheresis, neurologic condition is documented to deteriorate (for example, new or increase finger tip paresthesias or increased leg heaviness) upon stopping IVIG (or plasmapheresis)@
• Monoclonal gammopathy of undetermined significance (MUGS) (in which the pathogenesis of are thought to be the same as CIDP) will be allowed provided bone marrow aspirate and biopsy rules out multiple myeloma.
• Other immune mediated or suspected immune mediated neuropathies such as multifocal motor neuropathy or anti-myelin-associated glycoprotein (anti-MAG) neuropathy may be treated but will be analyzed and reported separately.

Exclusion Criteria

• Any evidence of hereditary cause for neuropathy that is known or likely hereditary demyelination neuropathy because of family history, foot deformity, mutilation of hands or feet, retinitis pigmentosa, ichthyosis, or liability to pressure palsies.
• Diphtheria, drug, or toxin exposure likely to be cause of neuropathy
• Conditions in which the pathogenesis of the neuropathy may be different from CIDP such as: Lyme disease (Borrelia burgdorferi infection), POEMS syndrome, Osteosclerotic myeloma, malignancies such as Waldenstrom macroglobulinemia, and Castleman's)
• Multiple myeloma
• HIV positive
• Insulin dependent Diabetes mellitus
• Chronic active hepatitis
• Age > 65 years old or 2.0.
• Liver cirrhosis, transaminases > 2 x of normal limits or bilirubin >2.0 unless due to Gilbert disease.
• Prior history of malignancy except localized basal cell or squamous skin cancer or other localized cancer considered cured only by surgery
• Positive pregnancy test, inability or unwillingness to pursue effective means of birth control, or failure to willingly accept or comprehend irreversible sterility as a side effect of therapy.
• Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible.
• Inability to give informed consent.
• Major hematological abnormalities such as platelet count less than 100,000/ul or absolute neutrophil count (ANC) less than 1000/ul.
• Failure to collect at least 2.0 x 106 cluster of differentiation 34 (CD34+) cells by apheresis and, if necessary, bone marrow harvest is a contraindication to treatment, i.e., receiving the conditioning regimen.