Table 1.
General characteristics of the randomized clinical trials that compared PBSCT versus BMT for the treatment of hematological malignancies
| Trial (reference) (no.) | Eligibility criteria | GVHD prophylaxisa | Patients characteristics | Disease |
Risk categoriesb |
|
|---|---|---|---|---|---|---|
| Favorable | Unfavorable | |||||
| Saudi Arabia (unpublished) (N = 83) | Age 15–50
Hematologic malignancies |
CSA/MTX (D+1,3,6) | Median age (range): 23 (15–48) | ALL = 18 | ||
| Donor HLA = sibling | AML = 29 | |||||
| Males (%):45 (54.2%) | CML = 28 | 42 | 39 | |||
| MDS = 8 | ||||||
| France (13) (N = 101) | Age <55 years
ALL, AML and CML (1st chronic phase), Donor HLA = sibling |
CSA/MTX (D+1,3,6) | Median age (range): 36 (16–53) | ALL = 19 | ||
| AML = 45 | ||||||
| CML = 37 | 95 | 6 | ||||
| Males (%):53 (52.5%) | MDS = 0 | |||||
| EBMT (12) (N = 350) | Age 16–55 years | CSA/MTX (D+1,3,6) | Median age (range): 38 (17–58) | ALL = 61 | ||
| De novo AML and ALL (in 1st or 2nd remission or in 1st incipient relapse), CML (in chronic or accelerate phase), MDS (except RAEBT) | AML = 126 | |||||
| CML = 152 | ||||||
| Males (%):196 (56.0%) | MDS = 11 | 301 | 38 | |||
| Donor HLA = sibling | ||||||
| Brazil (10) (N = 56) | Age 10–60 years | CSA/MTX (D+1,3,6,11) or CSA/PRED (3 pts at BMT arm) | Median age (range): 31 (7–60) | ALL = 5 | ||
| Hematological malignancies | AML = 11 | |||||
| Donor HLA = sibling | CML = 31 | |||||
| Males (%):38 (67.8%) | MDS = 5 | 37 | 19 | |||
| Others = 4 | ||||||
| Canada (11) (N = 228) | Age 16–65 years | CSA/MTX (D+1,3,6,11) | Median age (range): 45 (19–65) | ALL = 0 | ||
| CML (in chronic or accelerate phase), AML (in remission) and MDS | AML = 83 | |||||
| CML = 109 | ||||||
| Donor HLA = sibling | Males (%):133 (58.3%) | MDS = 36 | 169 | 53 | ||
| Norway (15) (N = 61) | Age 15–60 years
AML, ALL, CML, PMF and MDS |
CSA/MTX (D+1,3,6,11) | Median age (range): 42 (15–55) | ALL = 8 | ||
| Donor HLA = sibling or one mismatched family donor | AML = 24 | |||||
| Males (%):38 (62.3%) | CML = 26 | 42 | 12 | |||
| MDS = 1 | ||||||
| Others = 2 | ||||||
| UK (17) (N = 39) | Age 15–55 years
Any hematological malignancies |
CSA/MTX (D+1,3,6,11) | Median age (range): 37 (22–52) | ALL = 7 | ||
| Donor HLA = sibling | AML = 13 | |||||
| Males (%):29 (74.3%) | CML = 12 | 23 | 16 | |||
| MDS = 2 | ||||||
| Others = 5 | ||||||
| US1 (18) (N = 176) | Age 12–55 years | CSA/MTX (D+1,3,6,11) | Median age (range): 42 (12–56) | ALL = 22 | ||
| Hematological malignancies | AML = 39 | |||||
| Donor HLA = sibling | CML = 58 | 86 | 89 | |||
| Males (%): 122 (69.3%) | MDS = 16 | |||||
| Others = 39 | ||||||
| US2 (28)c (N = 18) | CML
Donor HLA = sibling |
CSA/MTX (D+1,3,6,11) | Median age (range): 46 (19–61) | |||
| CML = 18 | ||||||
| Males (%):12 (66.7%) | 14 | 4 | ||||
Abbreviations: ALL = acute lymphoblastic leukemia; AML = acute myeloid leukemia; BMT = bone marrow transplantation; Bu = busulfan; CML = chronic myeloid leukemia; CSA = cyclosporine A; Cy = cyclophosphamide; GVHD = graft-versus-host disease; MDS = myelodysplastic syndrome; MTX = methotrexate; NHL = non-hodgkin lymphoma; PBST = peripheral blood stem cell transplantation; PMF = primary myelofibrosis; Pred = prednisone; RAEBT = refractory anemia with excess blast in transformation; TBI = total body irradiation.
The dose of methotrexate used for GHVD prophylaxis was 15 mg/kg in D1 and 10 mg/kg in the rest of the days (D3, D6 and D11). Three patients in the Brazilian trial received prednisone. Initially, intravenous cyclosporine was used in all trials at a dose ranging from 2–5 mg/kg with a subsequent switch to oral administration according to blood levels.
Risk categorization was unavailable for 19 patients.
Seventy-two patients were included in this report, the majority of whom were in reference.18
Distribution of favorable/unfavorable groups: favorable (‘good’ prognosis): (chronic myelogenous leukemia (CML) in first chronic phase, acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) in first complete remission, and refractory anemia/refractory anemia with ringed sideroblasts subtypes of myelodysplastic syndromes (MDS)).
Unfavorable (‘poor’ prognosis): (CML in second chronic phase, accelerated phase or blast crisis; AML or ALL, refractory or in >first remission; refractory anemia with excess blasts or in transformation subtypes of MDS, multiple myeloma, Hodgkin’s disease, non-Hodgkin’s lymphoma, and idiopathic myelofibrosis).