A validated decision model for treating the anaemia of myelodysplastic syndromes with erythropoietin + granulocyte colony-stimulating factor: significant effects on quality of life.
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.
Hellström-Lindberg
Eva
E
Department of Medicine, Karolinska Institutet at Huddinge University Hospital, Stockholm, Sweden. Eva.Hellstrom-Lindberg@medhs.ki.se
Gulbrandsen
Nina
N
Lindberg
Greger
G
Ahlgren
Tomas
T
Dahl
Inger Marie S
IM
Dybedal
Ingunn
I
Grimfors
Gunnar
G
Hesse-Sundin
Eva
E
Hjorth
Martin
M
Kanter-Lewensohn
Lena
L
Linder
Olle
O
Luthman
Michaela
M
Löfvenberg
Eva
E
Oberg
Gunnar
G
Porwit-MacDonald
Anja
A
Rådlund
Anders
A
Samuelsson
Jan
J
Tangen
Jon Magnus
JM
Winquist
Ingemar
I
Wisloff
Finn
F
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
D004921:Erythropoietin; D016179:Granulocyte Colony-Stimulating Factor