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, H. Joachim Deeg

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Abstract

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 x 106 (range 0 to 410 x 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 x 109/L (100 000/μL) responded with a durable platelet count more than 100 x 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.

Original languageEnglish (US)
Pages (from-to)586-593
Number of pages8
JournalBlood
Volume98
Issue number3
DOIs
StatePublished - Aug 1 2001

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Primary Myelofibrosis
Stem cells
Platelets
Blood
Busulfan
Granulocyte Colony-Stimulating Factor
Transplantation
Recovery
Anthracyclines
Cytarabine
Platelet Count
Neutrophils
Toxicity
Blood Platelets
Hemoglobins
Peripheral Blood Stem Cells
Peripheral Blood Stem Cell Transplantation
Splenomegaly
Disease Progression
Survival

ASJC Scopus subject areas

  • Hematology

Cite this

Myeloablation and autologous peripheral blood stem cell rescue results in hematologic and clinical responses in patients with myeloid metaplasia with myelofibrosis. / Anderson, Jeanne E.; Tefferi, Ayalew; Craig, Fiona; Holmberg, Leona; Chauncey, Thomas; Appelbaum, Frederick R.; Guardiola, Philippe; Callander, Natalie; Freytes, Cesar; Gazitt, Yair; Razvillas, Betty; Joachim Deeg, H.

In: Blood, Vol. 98, No. 3, 01.08.2001, p. 586-593.

Research output: Contribution to journalArticle

Anderson, JE, Tefferi, A, Craig, F, Holmberg, L, Chauncey, T, Appelbaum, FR, Guardiola, P, Callander, N, Freytes, C, Gazitt, Y, Razvillas, B & Joachim Deeg, H 2001, 'Myeloablation and autologous peripheral blood stem cell rescue results in hematologic and clinical responses in patients with myeloid metaplasia with myelofibrosis', Blood, vol. 98, no. 3, pp. 586-593. https://doi.org/10.1182/blood.V98.3.586
Anderson, Jeanne E. ; Tefferi, Ayalew ; Craig, Fiona ; Holmberg, Leona ; Chauncey, Thomas ; Appelbaum, Frederick R. ; Guardiola, Philippe ; Callander, Natalie ; Freytes, Cesar ; Gazitt, Yair ; Razvillas, Betty ; Joachim Deeg, H. / Myeloablation and autologous peripheral blood stem cell rescue results in hematologic and clinical responses in patients with myeloid metaplasia with myelofibrosis. In: Blood. 2001 ; Vol. 98, No. 3. pp. 586-593.
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abstract = "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 x 106 (range 0 to 410 x 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 x 109/L (100 000/μL) responded with a durable platelet count more than 100 x 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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AU - Chauncey, Thomas

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