Hematopoietic Stem Cell Transplantation in Chronic Inflammatory Demyelinating Polyneuropathy

Inclusion criteria

• EMG definite CIDP according to the EFNS / EPS criteria (section 3.2.4c) AND
• Clinically typical CIDP (section 3.2.4 b) 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 either IVIG or plasmapheresis

• Failure to respond to therapy is defined by :

  1. 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

  2. Persistent dysphagia documented by either aspiration or insufficient clearing on video fluoroscopic examination.

     OR

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

     AND

  4. If patients are on IVIG or plasmapheresis, neurologic condition is documented to deteriorate (for example, new, increase fingertip parasthesias, 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.

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.
• Diptheria, drug, or toxin exposure likely to be cause of neuropathy
• Multifocal motor neuropathy
• Presence of sphincter disturbance
• 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 < 18 years old

• Significant end organ damage such as (not caused by CIDP):

  1. LVEF <40% or deterioration of LVEF during exercise test on MUGA or echocardiogram.
  2. Untreated life-threatening arrhythmia.
  3. Active ischemic heart disease or heart failure or myocardial infarction within the last 6 months
  4. DLCO <40% or FEV1/FEV < 50%
  5. Serum creatinine >2.0.
  6. 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.
• 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 ANC less than 1000/ul.
• Failure to collect at least 2.0 x 106 CD34+ cells by apheresis and, if necessary, bone marrow harvest is a contraindication to treatment, i.e., receiving the conditioning regimen.