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Blood, 15 October 2001, Vol. 98, No. 8, pp. 2326-2331
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Intensive chemotherapy followed by allogeneic or autologous stem
cell transplantation for patients with myelodysplastic
syndromes (MDSs) and acute myeloid leukemia following MDS
Theo de Witte,
Stefan Suciu,
Gregor Verhoef,
Boris Labar,
Eric Archimbaud,
Carlo Aul,
Dominique Selleslag,
Augustin Ferrant,
Pierre Wijermans,
Franco Mandelli,
Sergio Amadori,
Ulrich Jehn,
Petra Muus,
Marc Boogaerts,
Robert Zittoun,
Alois Gratwohl,
Heintz Zwierzina,
Anne Hagemeijer, and
Roel Willemze
From the University Medical Center St Radboud,
Nijmegen, The Netherlands; EORTC Data Center, Brussels, Belgium;
Departments of Hematology and Center for Human Genetics, University
Hospital Gasthuisberg, Leuven, Belgium; Clinical Hospital Rebro,
Zagreb, Croatia; Hôpital Edouard Herriot, Lyon, France; Heinrich
Heine University, Dusseldorf, Germany; St Jan's Hospital, Brugge,
Belgium; Clinique Universitaire St Luc, Brussels, Belgium; Leijenburg
Hospital, The Hague, The Netherlands; University La Sapienza, Roma,
Italy; Ospedale San Eugenio, Roma, Italy; Klinikum Grosshadern,
Ludwig-Maximilian University, Munich, Germany; Hôpital Dieu,
Paris, France; Kantonsspital, Basel, Switzerland; Medizinische Klinik,
Innsbruck, Austria; and Leiden University Medical Center, Leiden, The
Netherlands.
This study investigated the feasibility of allogeneic (alloSCT) and
autologous stem cell transplantation (ASCT) as postconsolidation therapy for patients with myelodysplastic syndromes (MDSs) or acute
myeloid leukemia after MDS. Patients with a histocompatible sibling
were candidates for alloSCT and the remaining patients for ASCT.
Remission-induction therapy consisted of 1 or 2 courses with
idarubicin, cytarabine, and etoposide, followed by one intensive consolidation course with cytarabine and mitoxantrone. Initially, bone
marrow cells were used for ASCT. Subsequently, mobilized blood stem
cells were used in an attempt to shorten posttransplantation hypoplasia. With a median follow-up of 3.6 years the 184 evaluable patients showed a 4-year survival rate of 26% and a median survival of
13 months. The remission-induction chemotherapy induced complete remission (CR) in 100 patients (54%). The 4-year disease-free survival
(DFS) rate was 29% and the median DFS was 12 months. Twenty-eight of
39 patients (72%) with a donor were allografted in CR-1, including 2 patients who underwent transplantation in CR-1 without a
consolidation course. Thirty-six of 59 patients (61%) without a donor
received ASCT in CR-1. The 4-year DFS rates in the group of patients
with or without a donor were 31% and 27%, respectively. The 4-year
survival rates from CR were 36% and 33%, respectively. This large
prospective study shows the feasibility of both alloSCT and ASCT. This
treatment approach leads to a relatively high remission rate, and the
majority of patients in remission received the SCT in CR-1. The ongoing
study investigates whether this approach is better than treatment with chemotherapy only.
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