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.