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High dose cyclophosphamide for severe refractory myasthenia gravis
  1. D E Gladstone1,
  2. T H Brannagan III2,
  3. R J Schwartzman3,
  4. A A Prestrud3,
  5. I Brodsky3
  1. 1Stony Brook University, Health Sciences Center, Division of Oncology, New York, USA
  2. 2Weill Medical College of Cornell University, New York, USA
  3. 3Drexel University College of Medicine, Philadelphia, USA
  1. Correspondence to:
 Dr D E Gladstone
 Stony Brook University, Health Sciences Center, Division of Oncology, T17-080, Stony Brook, NY 11794, USA; dsgladstonecomcast.net

https://doi.org/10.1136/jnnp.2003.019232

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    Myasthenia gravis (MG) exemplifies autoimmune disease. Most patients require immunomodulating treatment, including steroids, chemotherapy, or intravenous immunoglobulin (Ig), in addition to anticholinesterase treatment. Drachman et al1 published the beneficial effects of high dose cyclophosphamide in three patients with severe refractory myasthenia. We recount our experience of three myasthenic patients treated in a similar way.

    Materials and methods

    All patients participated in studies approved by the Drexel University College of Medicine and signed informed consent. These three patients with severe (class IVb) refractory MG includes all patients treated. Patients received cyclophosphamide 50 mg/kg (adjusted ideal body weight)/day over four consecutive days. Patients received antibacterial, antiviral, and antifungal prophylaxis. Haemorrhagic cystitis prophylaxis included Mesna and forced diuresis. Packed red cells and platelets were transfused to maintain haemoglobin ⩾8.5 g/dL and platelets ⩾10 × 109/L, respectively. Patients received filgrastion (G-CSF) (5 μg/kg/day) starting day 10 until their absolute neutrophil count (ANC) reached 10 × 109/L for two consecutive days.

    Results

    Patient 1 was diagnosed with seronegative MG at 30 years of age by a positive tensilon test and a decremental response on repetitive stimulation. Initial treatment included pyridostigine and plasmapheresis, but worsening symptoms prompted thymectomies at 12 and 18 months later. Her thymic pathology revealed thymic hyperplasia. Additional treatment with …

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