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Blood, 1 August 2001, Vol. 98, No. 3, pp. 586-593
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Myeloablation and autologous peripheral blood stem cell rescue
results in hematologic and clinical responses in patients with
myeloid metaplasia with myelofibrosis
Jeanne E. Anderson,
Ayalew Tefferi,
Fiona Craig,
Leona Holmberg,
Thomas Chauncey,
Frederick R. Appelbaum,
Philippe Guardiola,
Natalie Callander,
Cesar Freytes,
Yair Gazitt,
Betty Razvillas, and
H.
Joachim Deeg
From the Division of Hematology, Department of
Medicine, University of Texas Health Science Center at San Antonio;
South Texas Veterans Health Care System, Audie L. Murphy Division; both
of San Antonio, TX; Hematology Division, Mayo Clinic, Rochester, MN;
Clinical Research Division, Fred Hutchinson Cancer Research Center;
University of Washington School of Medicine; Seattle Veterans
Administration Hospital; both of Seattle, WA; and Hospital St Louis,
Paris, France.
Current therapeutic options for myeloid metaplasia with
myelofibrosis (MMM) are limited. A pilot study was conducted of
autologous peripheral blood stem cell (PBSC) collection in 27, followed
by transplantation in 21 patients with MMM. The median age was 59 (range 45-75) years. PBSCs were mobilized at steady state (n = 2),
after granulocyte colony-stimulating factor (G-CSF) alone (n = 17),
or after anthracycline-cytarabine induction plus G-CSF (n = 8). A
median of 11.6 × 106 (range 0 to
410 × 106) CD34+ cells per kilogram were
collected. Twenty-one patients then underwent myeloablation with oral
busulfan (16 mg/kg) and PBSC transplantation. The median times to
neutrophil and platelet recovery after transplantation were 21 (range
10-96) and 21 (range, 13 to  246) days, respectively. Five patients
received back-up PBSC infusion because of delayed neutrophil or
platelet recovery. The median follow-up is 390 (range 70-1623) days
after transplantation, and the 2-year actuarial survival is 61%. After
transplantion, 6 patients died: 3 of nonrelapse causes (1 within 100 days of PBSC infusion) and 3 of disease progression. Erythroid response
(hemoglobin 100 g/L [10 gm/dL] without transfusion for 8
weeks) occurred in 10 of 17 anemic patients. Four of 8 patients with a
platelet count less than 100 × 109/L (100 000/µL)
responded with a durable platelet count more than 100 × 109/L (100 000/µL). Symptomatic splenomegaly
improved in 7 of 10 patients. It is concluded that (1) PBSC collection
was feasible and stable engraftment occurred after transplantation in
most patients with MMM, (2) myeloablation with busulfan was associated with acceptable toxicity, (3) a significant proportion of patients derived clinical benefit after treatment, and (4) further investigation of this novel approach is warranted.
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