Drug treatment is superior to allografting as first-line therapy in chronic myeloid leukemia.
Early allogeneic hematopoietic stem cell transplantation (HSCT) has been proposed as primary treatment modality for patients with chronic myeloid leukemia (CML). This concept has been challenged by transplantation mortality and improved drug therapy. In a randomized study, primary HSCT and best available drug treatment (IFN based) were compared in newly diagnosed chronic phase CML patients. Assignment to treatment strategy was by genetic randomization according to availability of a matched related donor. Evaluation followed the intention-to-treat principle. Six hundred and twenty one patients with chronic phase CML were stratified for eligibility for HSCT. Three hundred and fifty four patients (62% male; median age, 40 years; range, 11-59 years) were eligible and randomized. One hundred and thirty five patients (38%) had a matched related donor, of whom 123 (91%) received a transplant within a median of 10 months (range, 2-106 months) from diagnosis. Two hundred and nineteen patients (62%) had no related donor and received best available drug treatment. With an observation time up to 11.2 years (median, 8.9 years), survival was superior for patients with drug treatment (P = .049), superiority being most pronounced in low-risk patients (P = .032). The general recommendation of HSCT as first-line treatment option in chronic phase CML can no longer be maintained. It should be replaced by a trial with modern drug treatment first.
Hehlmann
Rüdiger
R
III Medizinische Universitätsklinik, Medizinische Fakultät Mannheim der Universität Heidelberg, Wiesbadener Strasse 7-11, 63805 Mannheim, Germany. r.hehlmann@urz.uni-heidelberg.de
Berger
Ute
U
Pfirrmann
Markus
M
Heimpel
Hermann
H
Hochhaus
Andreas
A
Hasford
Joerg
J
Kolb
Hans-Jochem
HJ
Lahaye
Tanja
T
Maywald
Ole
O
Reiter
Andreas
A
Hossfeld
Dieter K
DK
Huber
Christoph
C
Löffler
Helmut
H
Pralle
Hans
H
Queisser
Wolfgang
W
Tobler
Andreas
A
Nerl
Christoph
C
Solenthaler
Max
M
Goebeler
Mariele E
ME
Griesshammer
Martin
M
Fischer
Thomas
T
Kremers
Stephan
S
Eimermacher
Hartmut
H
Pfreundschuh
Michael
M
Hirschmann
Wolf-Dietrich
WD
Lechner
Klaus
K
Wassmann
Barbara
B
Falge
Christiane
C
Kirchner
Hartmut H
HH
Gratwohl
Alois
A
D000293:Adolescent; D000328:Adult; D000970:Antineoplastic Agents / Q000494:pharmacology*; D002648:Child; D015331:Cohort Studies; D005260:Female; D006801:Humans; D015464:Leukemia, Myelogenous, Chronic, BCR-ABL Positive / Q000188:drug therapy* / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D012306:Risk; D014184:Transplantation, Homologous / Q000379:methods*; D016896:Treatment Outcome
D000970:Antineoplastic Agents