Study to Compare Busulfan-fludarabine With Thiotepa-fludarabine Regimen in Allogeneic Transplantation for Myelofibrosis

• Status: Completed
• Trial end date: 2016-12-31
• Target enrollment: 0
• Participant gender: All

Summary

This study will be performed as a prospective multicenter phase II trial for compare busulfan-fludarabine reduced-intensity conditioning (RIC) with thiotepa-fludarabine RIC regimen prior to allogeneic transplantation of hematopoietic cells for the treatment of myelofibrosis.

The primary endpoint for this study is to compare Progression Free Survival of two different RIC regimens for allogeneic stem cell transplantation in myelofibrosis.

Progression Free Survival is defined as the time from the date of randomization to the date of the first documented disease progression or relapse (according to the International Working Group Consensus Criteria) or death due to any cause. Patients who have neither progressed nor died at the time of study completion or who are lost to follow-up are censored at the data of the last follow up for progression of disease for this study.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Gruppo Italiano Trapianto di Midollo Osseo
• Treatments: Busulfan; Fludarabine; Fludarabine phosphate; Thiotepa; Vidarabine

Criteria

Inclusion Criteria:

• Age ≥ 18 ≤ 70 years

• Primary or secondary myelofibrosis after essential thrombocythemia or polycythemia vera

• One of the following unfavourable prognostic factors: Hb < 10 g/dL or leukocytes >25x109/L or > 1% circulating blasts in the peripheral blood or constitutional symptoms

• Performance Status (Karnofsky)≥ 60%

• Hematopoietic Cell Transplantation Comorbidity Score ≤ 5

• Written informed consent

Exclusion Criteria:

-≥ 20% blasts in peripheral blood and/or bone marrow

• Positive serologic markers for human immunodeficiency virus (HIV)

• Acute hepatitis B virus (HBV) or acute hepatic C virus (HCV) infection

• Severe irreversible renal, hepatic, pulmonary or cardiac disease, such as: --total bilirubin, Serum Glutamate Oxaloacetate Transaminase (SGOT) or Serum Glutamate Pyruvate Transaminase (SGPT) > 5 the upper normal limit;

• Left ventricular ejection fraction < 40%;

• Clearance creatinine < 30 ml/min;

• Diffusing Capacity of Lung for Carbon monoxide (DLCO) < 30% and/or receiving supplementary oxygen.

• Pregnancy or lactation

• Any active, uncontrolled infection

Donors:

• Age ≥ 18 < 65 years

• human leukocyte antigen (HLA)-identical sibling donor by high resolution DNA-based HLA-A, -B, -C, -DRB1, typing

• human leukocyte antigen (HLA)-identical unrelated donor by high resolution DNA-based human leukocyte antigen-A, human leukocyte antigen-B, human leukocyte antigen-C, human leukocyte antigen-DRB1 typing. One allele mismatched (class I) can be accepted for recipients up to 60 years.