Selected TMD patient characteristics
| . | All TMD patients . | TMD: observation only . | TMD: intervention . | Observation vs intervention, P . | ||||||
|---|---|---|---|---|---|---|---|---|---|---|
| n . | % . | Median (range) . | n . | % . | Median (range) . | n . | % . | Median (range) . | ||
| Total enrolled | 139 | |||||||||
| No. ineligible | 4 | 3 | ||||||||
| No. eligible | 135 | 97 | 106 | 78.5 | 29 | 21.5 | ||||
| Treatment arm at time of registration | ||||||||||
| Observation | 108 | 80.0 | 106 | 100 | 2 | 6.9 | – | |||
| Received TMD intervention later | 2 | 1.9 | – | |||||||
| Leukopheresis/exchange transfusion | 5 | 3.7 | 5 | 17.2 | – | |||||
| Chemotherapy (Ara-C) | 22 | 16.3 | 22 | 75.9 | – | |||||
| Age at diagnosis, d | 5 (0-58) | 6 (0-52) | 2 (0-58) | .061 | ||||||
| Age at study entry, d | 13 (1-66) | 14 (2-66) | 8 (1-61) | .004 | ||||||
| Follow-up time, d | 1153 (0-2857) | 1151 (0-2857) | 1196 (42-1923) | .978 | ||||||
| Time to development of AML/MDS, d | 21 | 16 | 441 (118-1085) | 17 | 16 | 444 (118-1085) | 4 | 14 | 280 (202-596) | .244 |
| Constitutional trisomy 21 | 113 | 83.7 | 87 | 82.1 | 26 | 89.7 | .407 | |||
| Trisomy 21 mosaicism | 22 | 16.3 | 19 | 17.9 | 3 | 10.3 | ||||
| Hepatomegaly | ||||||||||
| Not enlarged | 57 | 42.2 | 53 | 50.0 | 4 | 13.8 | < .001 | |||
| Enlarged but not below umbilcus | 63 | 46.7 | 49 | 46.2 | 14 | 48.3 | .845 | |||
| Below umbilicus | 15 | 11.1 | 4 | 3.8 | 11 | 37.9 | < .001 | |||
| Liver dysfunction (symptomatic) | ||||||||||
| Yes | 17 | 12.7 | 5 | 4.7 | 12 | 42.9 | < .001 | |||
| No | 117 | 87.3 | 101 | 95.3 | 16 | 57.1 | ||||
| Unknown | 1 | 0 | 1 | |||||||
| Organomegaly (liver or spleen) with respiratory compromise | ||||||||||
| Yes | 13 | 9.7 | 1 | 0.9 | 12 | 42.9 | < .001 | |||
| No | 121 | 90.3 | 105 | 99.1 | 16 | 57.1 | ||||
| Unknown | 1 | 0 | 1 | |||||||
| Diagnostic laboratory examination | ||||||||||
| WBC (× 103/μL) | 32.8 (4.8-259) | 26.9 (4.8-253) | 74.9 (9.4-259) | < .001 | ||||||
| A total blast count of ≥ 100,000/μL | ||||||||||
| Yes | 16 | 11.9 | 5 | 4.7 | 11 | 37.9 | < .001 | |||
| No | 119 | 88.1 | 101 | 95.3 | 18 | 62.1 | ||||
| Blasts in peripheral blood, % | 25 (0-92) | 22 (0-87) | 56 (0-92) | .002 | ||||||
| BM blasts, % | 15 (0-95) | 14 (0-78) | 27 (1-95) | .029 | ||||||
| HGB, g/dL | 14.8 (5-22.5) | 15.6 (5.0-22.5) | 11.9 (5.9-20.5) | < .001 | ||||||
| Platelet count (× 103/μL) | 125 (10-958) | 112 (10-958) | 182 (36-670) | .033 | ||||||
| . | All TMD patients . | TMD: observation only . | TMD: intervention . | Observation vs intervention, P . | ||||||
|---|---|---|---|---|---|---|---|---|---|---|
| n . | % . | Median (range) . | n . | % . | Median (range) . | n . | % . | Median (range) . | ||
| Total enrolled | 139 | |||||||||
| No. ineligible | 4 | 3 | ||||||||
| No. eligible | 135 | 97 | 106 | 78.5 | 29 | 21.5 | ||||
| Treatment arm at time of registration | ||||||||||
| Observation | 108 | 80.0 | 106 | 100 | 2 | 6.9 | – | |||
| Received TMD intervention later | 2 | 1.9 | – | |||||||
| Leukopheresis/exchange transfusion | 5 | 3.7 | 5 | 17.2 | – | |||||
| Chemotherapy (Ara-C) | 22 | 16.3 | 22 | 75.9 | – | |||||
| Age at diagnosis, d | 5 (0-58) | 6 (0-52) | 2 (0-58) | .061 | ||||||
| Age at study entry, d | 13 (1-66) | 14 (2-66) | 8 (1-61) | .004 | ||||||
| Follow-up time, d | 1153 (0-2857) | 1151 (0-2857) | 1196 (42-1923) | .978 | ||||||
| Time to development of AML/MDS, d | 21 | 16 | 441 (118-1085) | 17 | 16 | 444 (118-1085) | 4 | 14 | 280 (202-596) | .244 |
| Constitutional trisomy 21 | 113 | 83.7 | 87 | 82.1 | 26 | 89.7 | .407 | |||
| Trisomy 21 mosaicism | 22 | 16.3 | 19 | 17.9 | 3 | 10.3 | ||||
| Hepatomegaly | ||||||||||
| Not enlarged | 57 | 42.2 | 53 | 50.0 | 4 | 13.8 | < .001 | |||
| Enlarged but not below umbilcus | 63 | 46.7 | 49 | 46.2 | 14 | 48.3 | .845 | |||
| Below umbilicus | 15 | 11.1 | 4 | 3.8 | 11 | 37.9 | < .001 | |||
| Liver dysfunction (symptomatic) | ||||||||||
| Yes | 17 | 12.7 | 5 | 4.7 | 12 | 42.9 | < .001 | |||
| No | 117 | 87.3 | 101 | 95.3 | 16 | 57.1 | ||||
| Unknown | 1 | 0 | 1 | |||||||
| Organomegaly (liver or spleen) with respiratory compromise | ||||||||||
| Yes | 13 | 9.7 | 1 | 0.9 | 12 | 42.9 | < .001 | |||
| No | 121 | 90.3 | 105 | 99.1 | 16 | 57.1 | ||||
| Unknown | 1 | 0 | 1 | |||||||
| Diagnostic laboratory examination | ||||||||||
| WBC (× 103/μL) | 32.8 (4.8-259) | 26.9 (4.8-253) | 74.9 (9.4-259) | < .001 | ||||||
| A total blast count of ≥ 100,000/μL | ||||||||||
| Yes | 16 | 11.9 | 5 | 4.7 | 11 | 37.9 | < .001 | |||
| No | 119 | 88.1 | 101 | 95.3 | 18 | 62.1 | ||||
| Blasts in peripheral blood, % | 25 (0-92) | 22 (0-87) | 56 (0-92) | .002 | ||||||
| BM blasts, % | 15 (0-95) | 14 (0-78) | 27 (1-95) | .029 | ||||||
| HGB, g/dL | 14.8 (5-22.5) | 15.6 (5.0-22.5) | 11.9 (5.9-20.5) | < .001 | ||||||
| Platelet count (× 103/μL) | 125 (10-958) | 112 (10-958) | 182 (36-670) | .033 | ||||||
AML indicates acute myeloid leukemia; HGB, hemoglobin; MDS, myelodysplastic syndromes; TMD, transient myeloproliferative disorder; and WBC, white blood cell.