title: Oral cladribine for B-cell chronic lymphocytic leukaemia: report of a phase II trial with a 3-d, 3-weekly schedule in untreated and pretreated patients, and a long-term follow-up of 126 previously untreated patients.
abstract: A phase II study was undertaken to evaluate the efficacy and toxicity of a new schedule of cladribine administration (10 mg/m2 orally daily for 3 d every 3 weeks) in 107 patients with B-cell chronic lymphocytic leukaemia (CLL). To minimize toxicity, treatment withdrawal criteria were defined. The results of the 63 previously untreated patients were retrospectively compared with 63 from an earlier study using a 5-d monthly schedule. The compiled data were analysed for prognostic factors for survival. No significant difference regarding response were seen in the two cohorts of the 126 previously untreated patients. The complete response (CR), nodular partial response (nPR) and partial response (PR) rates were 15%, 21% and 41%. Quality of response had no impact on survival. The 3- and 5-year overall survival for previously untreated patients was 73% and 58%, respectively, with a median follow-up of 54 months. Pretreatment haemoglobin <11.0 g/dl and elevated beta-2-microglobulin had a negative influence on survival. Major infections occurred in 21% of patients in the 3-d study compared with 35% in the 5-d study. The overall response (OR) and CR rates in the 40 previously treated patients were 34% and 5% respectively. Median overall survival was 24 months and median progression-free survival for responding patients was 14 months. Cladribine used as a single agent is an effective treatment with an acceptable safety profile for pretreated and untreated B-CLL. The achievement of complete remission was not a prerequisite for long-term survival.
authors.affiliation: Department of Haematology, University Hospital, Linköping, Sweden. karin.karlsson@lio.se
authors.forename: Karin
authors.initials: K
authors.lastname: Karlsson
authors.forename: Mats
authors.initials: M
authors.lastname: Strömberg
authors.forename: Jan
authors.initials: J
authors.lastname: Liliemark
authors.forename: André
authors.initials: A
authors.lastname: Delannoy
authors.forename: S A N
authors.initials: SA
authors.lastname: Johnson
authors.forename: Anja
authors.initials: A
authors.lastname: Porwit
authors.forename: Eva
authors.initials: E
authors.lastname: Kimby
authors.forename: Gerd
authors.initials: G
authors.lastname: Lärfars
authors.forename: Ilse
authors.initials: I
authors.lastname: Cristiansen
authors.forename: Göran
authors.initials: G
authors.lastname: Nilsson
authors.forename: Fredrik
authors.initials: F
authors.lastname: Celsing
authors.forename: Gunnel
authors.initials: G
authors.lastname: Sundström
authors.forename: Mikaela
authors.initials: M
authors.lastname: Luthman
authors.forename: Ulf
authors.initials: U
authors.lastname: Tidefelt
authors.forename: Jonas
authors.initials: J
authors.lastname: Wallvik
authors.forename: Gunnar
authors.initials: G
authors.lastname: Juliusson
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000367:Age Factors; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D017338:Cladribine / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D018450:Disease Progression; D018572:Disease-Free Survival; D004334:Drug Administration Schedule; D005500:Follow-Up Studies; D006801:Humans; D015451:Leukemia, Lymphocytic, Chronic, B-Cell / Q000188:drug therapy*; D008875:Middle Aged; D018365:Neoplasm, Residual; D009894:Opportunistic Infections / Q000139:chemically induced; D011379:Prognosis; D012189:Retrospective Studies; D015996:Survival Rate; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D017338:Cladribine