title: [Efficacy and safety of low-dose cyclophosphamide plus corticosteroids for type I/II myasthenia gravis].
abstract: OBJECTIVE
  To evaluate the efficacy and safety of low-dose cyclophosphamide plus corticosteroids for type I/II myasthenia gravis (MG).
  
  
  METHODS
  This trial was prospective, non-random and open-labeled. We selected 160 patients with steroid-insensitive MG from January 1999 to October 2011. Each patient received an oral dose of prednisone 0.5 mg×kg(-1)×d(-1), cyclophosphamide 8 mg×kg(-1)×time(-1) once weekly intravenously and oral pyridostigmine 36 mg×kg(-1)×d(-1). The efficacies were assessed by absolute and relative MG scores. The clinical treatment cycle was 30 weeks.
  
  
  RESULTS
  (1) Among them, 74/106 type I MG patients (69.9%) reached clinical relative scores ≥ 95% in 30 weeks. The total dose of cyclophosphamide was 12 g. And 35/54 type II MG patients (64.8%) reached clinical relative scores ≥ 95% in 30 weeks. No statistically significant difference existed between two groups (P = 0.521). (2) All patients had various degrees of improvement in 30 weeks. The difference was statistically significant by Mann-Whitney U test (P ≤ 0.05). (3) There were minor side effects in these all patients. (4) When the total dose of cyclophosphamide reached 4 g, the cure rate in type I patients was higher than that of in type II patients (P = 0.000). When the total dose of cyclophosphamide reached 12 g, the cure rate in type II patients was higher than that of in type I patients (P = 0.001). (5) The cure dose of cyclophosphamide was 4-8 g in 58.4% of type I patients versus 8 - 12 g in 61.1% of type II patients.
  
  
  CONCLUSIONS
  The combined treatment of low-dose cyclophosphamide and corticosteroids in glucocorticoid-insensitive type MG (type I/II) is both effective and safe. And the sensitivity to cyclophosphamide varies for different clinical types. It is higher in type I than type II patients.
authors.affiliation: Department of Neurology, Sun Yat-sen University, Guangzhou, China.
authors.forename: Hui-yu
authors.initials: HY
authors.lastname: Feng
authors.forename: Wei-bin
authors.initials: WB
authors.lastname: Liu
authors.forename: Xin
authors.initials: X
authors.lastname: Huang
authors.forename: Li
authors.initials: L
authors.lastname: Qiu
authors.forename: Yan
authors.initials: Y
authors.lastname: Li
authors.forename: Hai-yan
authors.initials: HY
authors.lastname: Wang
authors.forename: Chuan-ming
authors.initials: CM
authors.lastname: Luo
mesh_terms: D000293:Adolescent; D000305:Adrenal Cortex Hormones / Q000009:adverse effects / Q000627:therapeutic use*; D000328:Adult; D000368:Aged; D003520:Cyclophosphamide / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009157:Myasthenia Gravis / Q000188:drug therapy*; D011446:Prospective Studies; D055815:Young Adult
chemical_list: D000305:Adrenal Cortex Hormones; D003520:Cyclophosphamide