Comparison of Intensive Chemotherapy and Hypomethylating Agents before Allogeneic Stem Cell Transplantation for Advanced Myelodysplastic Syndromes: A Study of the Myelodysplastic Syndrome Subcommittee of the Chronic Malignancies Working Party of the European Society for Blood and Marrow Transplant Research.
The European Society for Blood and Marrow Transplant Research data set was used to retrospectively analyze the outcomes of hypomethylating therapy (HMA) compared with those of conventional chemotherapy (CC) before hematopoietic stem cell transplantation (HSCT) in 209 patients with advanced myelodysplastic syndromes. Median follow-up was 22.1 months and the median age of the group was 57.6 years with 37% of the population older than > 60 years. The majority of patients (59%) received reduced-intensity conditioning and 34% and 27% had intermediate-2 and high international prognostic scoring system (IPSS) scores. At time of HSCT, 32% of patients did not achieve complete remission (CR) and 13% had primary refractory disease. On univariate analysis, outcomes at 3 years were not significantly different between HMA and CC for overall survival (OS), relapse-free survival (RFS), cumulative incidence of relapse (CIR), and nonrelapse mortality (NRM): OS (42% versus 35%), RFS (29% versus 31%), CIR (45% versus 40%), and NRM (26% versus 28%). Comparing characteristics of the groups, there were more patients < 55 years old, more patients in CR (68% versus 32%), and fewer patients with primary refractory disease in the CC group than in the HMA group (10% versus 19%, P < .001). Patients with primary refractory disease had worse outcomes than those in CR with regard to OS (hazard ratio [HR], 2.42; 95% confidence interval [CI], 1.41 to 4.13; P = .001), RFS (HR, 2.27; 95% CI, 1.37 to 3.76; P = .001), and NRM (HR, 2.49; 95% CI, 1.18 to 5.26; P = .016). In addition, an adverse effect of IPSS-R cytogenetic risk group was evident for RFS. In summary, outcomes after HSCT are similar for patients receiving HMA compared with those receiving CC, despite the higher proportion of patients with primary refractory disease in the HMA group.
Department of Haematological Medicine, Kings College Hospital, London, United Kingdom. Electronic address: victoriapotter@nhs.net.
Victoria T
VT
Potter
Centro Interdipartimentale di Biostatistica e Bioinformatica, Università Tor Vergata, Roma, Italy.
Simona
S
Iacobelli
European Society for Blood and Marrow Transplant Research Data Office, Leiden University Medical Centre, Leiden, Netherlands.
Anja
A
van Biezen
Division of Hematology, Uz Gasthuisberg, Leuven, Belgium.
Johann
J
Maertens
Division of Hematology, Institut Gustave Roussy, Villejuif, France.
Jean-Henri
JH
Bourhis
Division of Hematology, University Hospital of Basel, Basel, Switzerland.
Jakob R
JR
Passweg
Hôpital HURIEZ, Lille, France.
Ibrahim
I
Yakhoub-Agha
Division of Hematology, CHU, Bordeaux, France.
Reza
R
Tabrizi
Service de Thérapie Cellulaire et d'hématologie clinique adulte, Hotel-Dieu, Clermont-Ferrand, France.
Jacques-Olivier
JO
Bay
Division of Hematology, CHU, Nantes, France.
Patrice
P
Chevallier
Division of Hematology, HUG, Geneva, Switzerland.
Yves
Y
Chalandon
Hopital de Purpan, Toulouse, France.
Anne
A
Huynh
Hématologie Clinique, Hospital A. Michallon, Grenoble, France.
Jean Yves
JY
Cahn
Department of Hematology, Karolinska University Hospital, Stockholm, Sweden.
Per
P
Ljungman
Centre for Clinical Haematology, Queen Elizabeth Hospital, Birmingham, United Kingdom.
Charles
C
Craddock
Department of Hematology, Skanes University Hospital, Lund, Sweden.
Stig
S
Lenhoff
Nottingham City Hospital, Nottingham, United Kingdom.
N H
NH
Russell
Départment d'Hématologie Clinique, Montpellier, France.
Nathalie
N
Fegueux
Department of Hematologie-BMT, Hospital St. Louis, Paris, France.
Gerard
G
Socié
S.S.C.V.D. Trapianto di Cellule Staminali, A.O.U. Citta della Salute e della Scienza di Torino Presidio Molinette, Torino, Italy.
Benedetto
B
Bruno
Department of Hematology, VU University Medical Center, Amsterdam, Netherlands.
Ellen
E
Meijer
Department of Haematological Medicine, Kings College London, United Kingdom.
G J
GJ
Mufti
Department of Tumorimmunology Radboud University Medical Centre, Nijmegen, Netherlands.
Theo
T
de Witte
Division of Hematology - Bone Marrow Transplantation, Saint-Louis Hospital, Paris, France.
Marie
M
Robin
Department of Stem Cell Transplantation University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Nicolaus
N
Kröger
D000328:Adult; D000368:Aged; D000964:Antimetabolites, Antineoplastic / Q000592:standards / Q000627:therapeutic use*; D000970:Antineoplastic Agents / Q000592:standards / Q000627:therapeutic use*; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods / Q000401:mortality / Q000592:standards; D006801:Humans; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000401:mortality; D012074:Remission Induction; D012189:Retrospective Studies; D016879:Salvage Therapy; D016019:Survival Analysis; D055815:Young Adult
D000964:Antimetabolites, Antineoplastic; D000970:Antineoplastic Agents
Azacitidine; Chemotherapy; Myelodysplastic syndrome; Stem cell transplantation