title: Long-term outcome of patients with acquired primary idiopathic pure red cell aplasia receiving cyclosporine A. A nationwide cohort study in Japan for the PRCA Collaborative Study Group.
abstract: OBJECTIVE
  Cyclosporine A (CsA) has become one of the leading agents for the treatment of pure red cell aplasia (PRCA). However, further studies are necessary to determine the relapse-free survival (RFS) and overall survival (OS) of patients treated with this drug, the minimum duration of therapy for induction of remission, and whether or not there is need for maintenance treatment.
  
  
  METHODS
  We conducted a nationwide survey in Japan. From a total of 185 patients (with 73 primary idiopathic PRCA and 112 with secondary PRCA), we evaluated 62 patients with primary idiopathic PRCA for this report.
  
  
  RESULTS
  The remission induction therapy for these patients included CsA (n=31), corticosteroids (CS) (n=20) or other drugs (n=11). CsA and CS produced remissions in 23 (74%) and 12 (60%) patients, respectively. The salvage treatment produced remissions in 58 patients (94%). Forty-one and 15 patients were maintained on CsA+/-CS (CsA-containing group) or CS alone (CS group), respectively. The median RFS in the CsA-containing group was 103 months, longer than that seen in the CS group (33 months) (p<0.01). Of 14 patients whose CsA was discontinued, 12 patients (86%) relapsed after a median of 3 months (range 1.5 to 40 months), while only 3 of 27 patients (11%) relapsed during CsA-containing maintenance therapy. Thus, the discontinuance of maintenance therapy was strongly correlated with relapse (p<0.001). Four patients in the CsA-containing group died; however, the OS of this group was not significantly different from that of the CS-groups (p=0.104).
  
  
  CONCLUSIONS
  CsA-containing regimens sustain prolonged RFS more effectively than CS in primary idiopathic PRCA and seem to be important to prevent relapse.
authors.affiliation: Division of Haematologyand Oncology, Department of Medicine,Akita University School of Medicine,1-1-1 Hondo, Akita, Japan. ksawada@doc.med.akita- u.ac.jp
authors.forename: Ken-ichi
authors.initials: K
authors.lastname: Sawada
authors.forename: Makoto
authors.initials: M
authors.lastname: Hirokawa
authors.forename: Naohito
authors.initials: N
authors.lastname: Fujishima
authors.forename: Masanao
authors.initials: M
authors.lastname: Teramura
authors.forename: Masami
authors.initials: M
authors.lastname: Bessho
authors.forename: Kazuo
authors.initials: K
authors.lastname: Dan
authors.forename: Hisashi
authors.initials: H
authors.lastname: Tsurumi
authors.forename: Shinji
authors.initials: S
authors.lastname: Nakao
authors.forename: Akio
authors.initials: A
authors.lastname: Urabe
authors.forename: Mitsuhiro
authors.initials: M
authors.lastname: Omine
authors.forename: Keiya
authors.initials: K
authors.lastname: Ozawa
mesh_terms: D000293:Adolescent; D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001327:Autoimmune Diseases / Q000188:drug therapy* / Q000401:mortality; D015331:Cohort Studies; D003520:Cyclophosphamide / Q000627:therapeutic use; D016572:Cyclosporine / Q000627:therapeutic use*; D018572:Disease-Free Survival; D004341:Drug Evaluation; D005260:Female; D005500:Follow-Up Studies; D006306:Health Surveys; D019337:Hematologic Neoplasms / Q000150:complications; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007564:Japan / Q000453:epidemiology; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000150:complications; D011289:Preleukemia / Q000150:complications; D012008:Recurrence; D012010:Red-Cell Aplasia, Pure / Q000188:drug therapy* / Q000209:etiology / Q000401:mortality; D012074:Remission Induction; D016879:Salvage Therapy; D016019:Survival Analysis; D016896:Treatment Outcome
chemical_list: D000305:Adrenal Cortex Hormones; D007166:Immunosuppressive Agents; D016572:Cyclosporine; D003520:Cyclophosphamide