Clinical trial NCT00599547: Allogeneic Stem Cell Transplantation (SCT) After Dose-reduced Conditioning for Myelofibrosis Patients
Official title: Allogeneic Stem Cell Transplantation After Dose-reduced Intensity Conditioning Regimen for Patients With Myelofibrosis With Myeloid Metaplasia (MMM): A Phase II-study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Myelofibrosis
Keywords: Hematopoietic Stem Cell Transplantation
Lead sponsor: Universitätsklinikum Hamburg-Eppendorf
Sponsor class: OTHER
First posted: 2008-01-23
Enrollment: 106
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine whether a reduced intensity conditioning regimen followed by allogeneic stem cell transplantation is a feasible and effective treatment for patients with primary myelofibrosis.

Detailed description: The purpose of this study is to determine whether a reduced intensity conditioning regimen followed by allogeneic stem cell transplantation from related or unrelated donors is a feasible and effective treatment for patients with primary myelofibrosis.

Eligibility criteria: Inclusion Criteria:

* Disease: Histologically proven Myelofibrosis with myeloid metaplasia/MMM defined as per the Italian Consensus Conference on MMM diagnosis with either primary MMM or secondary to Polycythaemia vera or essential thrombocythaemia with

  * "intermediate risk" or "high risk" score according to the Lille Scoring System
  * OR "low risk" score according to the Lille Scoring System with constitutional symptoms (fever, night sweat, weight loss \> 5% of body weight)
  * OR "high risk" score according to the Cervantes score
* Age:

  * Patients between 50 and 70 years of age and no limited life expectancy for other reasons than MMM
  * Patients under 50 years of age who are not eligible for a standard myeloablative conditioning, (e.g., because of prior autologous stem cell, history of severe infections like fungal infections, history of cardiac toxicity or other reasons according to the treating physician)
* HLA compatible or identical donor, related or unrelated (one mismatch allowed)
* Written informed consent of the patient

Exclusion Criteria:

* Positive search for bcr-abl on blood.
* Serious irreversible renal, hepatic, pulmonary or cardiac disease
* Central nervous involvement
* Positive serology for HIV
* Pregnant or lactating women
* Patients with a life-expectancy of less than six months because of another debilitating disease
* Serious psychiatric or psychological disorders

Primary outcomes:
measure: Transplant-related mortality by day +100 and by one year post-transplant, hematopoietic recovery by day +100 post-transplant, myeloid and T-cell chimerism by day +100 posttransplant, toxicity of the regimen acc. to Bearman scale
timeFrame: Follow-up until day +1095
Secondary outcomes:
measure: VOD-Staging and VOD-Grading acc. to the McDonald-scale, incidence of GvHD, overall survival post-transplant, disease-free survival post-transplant
timeFrame: Follow-up until day +1095
Other outcomes:
Locations:
city: Hamburg
country: Germany
facility: University Medical Center Hamburg-Eppendorf
Study officials:
affiliation: Universitätsklinikum Hamburg-Eppendorf
name: Nicolaus Kroeger, Prof. Dr.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Kroger N, Holler E, Kobbe G, Bornhauser M, Schwerdtfeger R, Baurmann H, Nagler A, Bethge W, Stelljes M, Uharek L, Wandt H, Burchert A, Corradini P, Schubert J, Kaufmann M, Dreger P, Wulf GG, Einsele H, Zabelina T, Kvasnicka HM, Thiele J, Brand R, Zander AR, Niederwieser D, de Witte TM. Allogeneic stem cell transplantation after reduced-intensity conditioning in patients with myelofibrosis: a prospective, multicenter study of the Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation. Blood. 2009 Dec 17;114(26):5264-70. doi: 10.1182/blood-2009-07-234880. Epub 2009 Oct 7.