title: High-dose cyclophosphamide results in long-term disease remission with restoration of a normal quality of life in patients with severe refractory chronic inflammatory demyelinating polyneuropathy.
abstract: We previously reported the improvement of clinical parameters in severe, refractory chronic inflammatory demyelinating polyneuropathy (CIDP) following high-dose cyclophosphamide therapy. Here, we examine effects of this therapy on quality of life, report long-term clinical follow-up, and include new data on a fifth patient. Patients completed The Medical Outcomes Short Form 36 to assess quality of life impact. Pretherapy and post-therapy scores were compared with the Wilcoxon Signed Ranks Test. Patient's post-therapy scores were compared with the normal United States population. Functional status was assessed with the Modified Rankin score. Strength was assessed using a summated MRC strength. Nerve conduction studies were conducted using standard techniques with supramaximal stimulation. The median follow-up for the five patients is 2.9 (range: 1.6-4.8) years. The first four patients remain off all immunomodulatory medications. In six of the eight quality of life scales measured, patients enjoyed clinically significant improvement. Their overall strength increased by a median change of 10 (range: -1 to 20); their overall Modified Rankin score increased by a median of 3 (range: 0-4), and their summated compound motor action potential amplitudes increased by a mean of 3.69 mV (range: 0.156-7.83). Patient 5 has had stabilization of motor strength. High-dose cyclophosphamide can markedly improve functionality and quality of life for patients with severe refractory CIDP.
authors.affiliation: Stony Brook University, Long Island Cancer Center, Stony Brook, NY 11794, USA. dgladstone@notes.cc.sunysb.edu
authors.forename: Douglas E
authors.initials: DE
authors.lastname: Gladstone
authors.forename: Ann A
authors.initials: AA
authors.lastname: Prestrud
authors.forename: Thomas H
authors.initials: TH
authors.lastname: Brannagan
mesh_terms: D000328:Adult; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D009431:Neural Conduction / Q000187:drug effects; D020277:Polyradiculoneuropathy, Chronic Inflammatory Demyelinating / Q000188:drug therapy*; D011788:Quality of Life; D020127:Recovery of Function; D012074:Remission Induction
chemical_list: D007166:Immunosuppressive Agents; D003520:Cyclophosphamide