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 - Research report

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.