title: A validated decision model for treating the anaemia of myelodysplastic syndromes with erythropoietin + granulocyte colony-stimulating factor: significant effects on quality of life.
abstract: We have published previously a prototype of a decision model for anaemic patients with myelodysplastic syndromes (MDS), in which transfusion need and serum erythropoietin (S-Epo) were used to define three groups with different probabilities of erythroid response to treatment with granulocyte colony-stimulating factor (G-CSF) + Epo. S-Epo </= 500 U/l and a transfusion need of < 2 units/month predicted a high probability of response to treatment, S-Epo > 500 U/l and >/= 2 units/month for a poor response, whereas the presence of only one negative prognostic marker predicted an intermediate response. A total of 53 patients from a prospective study were included in our evaluation sample. Patients with good or intermediate probability of response were treated with G-CSF + Epo. The overall response rate was 42% with 28.3% achieving a complete and 13.2% a partial response to treatment. The response rates were 61% and 14% in the good and intermediate predictive groups respectively. The model retained a significant predictive value in the evaluation sample (P < 0.001). Median duration of response was 23 months. Scores for global health and quality of life (QOL) were significantly lower in MDS patients than in a reference population, and fatigue and dyspnoea was significantly more prominent. Global QOL improved in patients responding to treatment (P = 0.01). The validated decision model defined a subgroup of patients with a response rate of 61% (95% confidence interval 48-74%) to treatment with G-CSF + Epo. The majority of these patients have shown complete and durable responses.
authors.affiliation: Department of Medicine, Karolinska Institutet at Huddinge University Hospital, Stockholm, Sweden. Eva.Hellstrom-Lindberg@medhs.ki.se
authors.forename: Eva
authors.initials: E
authors.lastname: Hellström-Lindberg
authors.forename: Nina
authors.initials: N
authors.lastname: Gulbrandsen
authors.forename: Greger
authors.initials: G
authors.lastname: Lindberg
authors.forename: Tomas
authors.initials: T
authors.lastname: Ahlgren
authors.forename: Inger Marie S
authors.initials: IM
authors.lastname: Dahl
authors.forename: Ingunn
authors.initials: I
authors.lastname: Dybedal
authors.forename: Gunnar
authors.initials: G
authors.lastname: Grimfors
authors.forename: Eva
authors.initials: E
authors.lastname: Hesse-Sundin
authors.forename: Martin
authors.initials: M
authors.lastname: Hjorth
authors.forename: Lena
authors.initials: L
authors.lastname: Kanter-Lewensohn
authors.forename: Olle
authors.initials: O
authors.lastname: Linder
authors.forename: Michaela
authors.initials: M
authors.lastname: Luthman
authors.forename: Eva
authors.initials: E
authors.lastname: Löfvenberg
authors.forename: Gunnar
authors.initials: G
authors.lastname: Oberg
authors.forename: Anja
authors.initials: A
authors.lastname: Porwit-MacDonald
authors.forename: Anders
authors.initials: A
authors.lastname: Rådlund
authors.forename: Jan
authors.initials: J
authors.lastname: Samuelsson
authors.forename: Jon Magnus
authors.initials: JM
authors.lastname: Tangen
authors.forename: Ingemar
authors.initials: I
authors.lastname: Winquist
authors.forename: Finn
authors.initials: F
authors.lastname: Wisloff
mesh_terms: D000368:Aged; D000740:Anemia / Q000628:therapy*; D000753:Anemia, Refractory / Q000628:therapy; D000754:Anemia, Refractory, with Excess of Blasts / Q000628:therapy; D000756:Anemia, Sideroblastic / Q000628:therapy; D001803:Blood Transfusion; D003661:Decision Support Techniques; D004921:Erythropoietin / Q000627:therapeutic use*; D005260:Female; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000628:therapy*; D011379:Prognosis; D011446:Prospective Studies; D011788:Quality of Life; D016896:Treatment Outcome
chemical_list: D004921:Erythropoietin; D016179:Granulocyte Colony-Stimulating Factor