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EBMT transplant-specific risk score for MDS
Results
Patients
The characteristics of the patients and transplants of the total EBMT cohort (n=1059) are listed in Table 1. The median follow-up was 69 months (95% CI: 62-76) and the 5-year overall survival was 38.6% (95% CI: 35.4-41.7) while the relapse-free survival was 33.5% (95% CI: 30.4- 36.6).
Transplant-specific risk score
Table 2 summarizes the seven variables identified as independent predictors of overall survival in the multivari- able analysis: age ≥50 years, matched unrelated donor, Karnofsky Performance Status <90%, very poor cytogenet- ics or monosomal karyotype, positive cytomegalovirus (CMV) status of the recipient, blood blasts >1%, and platelet count ≤50 x 109/L at the time of transplantation. A weighted score of two was assigned to older age (≥50 years) and very poor cytogenetics or monosomal karyotype, whereas matched unrelated donor, Karnofsky Performance Status <90%, positive CMV status of the recipient, blood
Table 1. Patient and transplantation characteristics of 1059 patients with myelodysplastic syndromes in the total EBMT cohort.
blasts >1%, and platelet count ≤50 x 109/L prior to trans- plantation were assigned a score of one.
The overall score ranged from zero to nine, with increas- ing scores indicating higher risk of death. Based on the score, four risk groups were delineated: low (0-1), interme- diate (2-3), high (4-5), and very high (>5). The hazard ratio for death (with the low-risk group as the reference) was 2.02 (95% CI: 1.41-2.90) for the intermediate-risk group, 3.49 (95% CI: 2.45-4.97) for the high-risk group, and 5.90 (95% CI: 4.01-8.67) for the very high–risk group. Corresponding survival rates were 68.7% for the low-risk group, 43.2% for the intermediate-risk group, 26.6% for the high-risk group, and 9.5% for the very high-risk group. Overall, the EBMT transplant-specific risk score was predic- tive of overall survival (P<0.001) (Figure 1).
Secondary endpoints
The EBMT cohort was also used to apply the developed score to all secondary objectives. The developed score (overall and in all risk groups) was associated with all sec- ondary endpoints (P=0.001) (Table 3 and Figure 1B-D). The 5-year relapse-free survival rate was 68.4% (95% CI:
Characteristics
Number of patients
Age at transplant, years
median (range)
Patient’s sex female male
Secondary disorder no
yes
unknown
Karnofsky index, % 90 to 100
<90
unknown
Comorbidity index, % 0to2
≥3
unknown
Cytogenetic risk very good good intermediate poor
very poor monosomal karyotype
Marrow blasts at transplant, % ≤2
2to5 5to10 >10 unknown
Blood blasts at transplant, % ≤1
>1
Total cohort, n (%)
1059
56 (18 to 73)
446 (42) 613 (58)
776 (74) 201 (19) 92 (7)
630 (60) 252 (23) 177(17)
462 (44) 190 (18) 407 (38)
26 (3) 132 (13) 357 (34) 299 (28) 45 (4) 200 (19)
333 (31) 258 (24) 170 (16) 114 (12) 210 (17)
929 (88) 130 (12)
Platelets at transplant, x 109/L ≤50
>50
Revised IPSS category very low
low
intermediate
high very high unknown
Conditioning intensity myeloablative reduced non-myeloablative
Pretreatment chemotherapy hypomethylating agents both
none
Graft source bone marrow peripheral blood
Time to transplant, months median (range)
Donor type HLA-identical sibling matched unrelated
Cytomegalovirus serostatus of recipient negative
positive
unknown
Anti-thymocyte globulin
Ex vivo T-cell depletion
n: number; IPSS: International Prognosis Scoring System.
382 (38)
677 (62)
32 (3) 259 (24) 317 (32) 205 (20) 86 (8) 150 (13)
336 (32) 620 (58) 103 (10)
235 (22) 214 (20) 42 (4) 568 (54)
147 (14)
912 (86)
7 (0.2 to 328)
622 (59)
437 (41)
416 (39) 614 (58) 29 (3)
491 (46)
43 (4)
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