Demographic and disease characteristics at diagnosis, incidence of biologic markers, treatment, and clinical outcomes of polycythemic children
| Clinical and hematologic characteristics . | SP (n = 11) . | HP (n = 3) . |
|---|---|---|
| Male sex/female sex | 8/3 | 1/2 |
| Median age, y (range) | 16 (6-19) | 11 (11-14) |
| Median WBCs, × 109/L, (range) | 6.73 (4.86-28.67) | 6.76 (5.4-6.77) |
| Median Hct, % (range) | 53.9 (50-72.5) | 53.2 (42.4-54.5) |
| Median platelets, × 109/L, (range) | 217 (166-945) | 207 (202-271) |
| Symptoms, n (%) | 5/11 (45.5%) | 0 |
| Headache | 4/5 | |
| Paresthesias | 1/5 | |
| Splenomegaly, n (%) | 3/11 (27%) | 0/3 |
| Thrombotic events before/at diagnosis, % | 0 | 0 |
| Bleedings before/at diagnosis, % | 0 | 0 |
| Epo serum level, (range) | 4.5 (1.1-16.4) | 4.5; 5; 7.4 |
| BM biopsy, n (%) | 10/11 | 1/3 |
| Reticulin fibrosis 0-1/1-2, n (%) | 3/10 (30%) | 1/1 |
| Biologic markers | (n = 11) | (n = 3) |
| EEC positive, n (%) | 4/10 (40%) | 0/3 |
| JAK2V617, n (%) | 3/11 (27%) | 0/3 |
| V617F allele burden | 39%; 43.5%; 48% | |
| Clonal hemopoiesis, no. females, % | 2/3 | 0/2 |
| Treatment | (n = 11) | (n = 3) |
| Antiplatelet treatment, n (%) | 4/11 (36 %) | 0 |
| Cytoreductive therapy, n (%) | 8/11 (72%) | 1/3 (33%) |
| Phlebotomy or erythrocytapheresis | 6/8 (75%) | 1/3 |
| Phlebotomy + HU | 2/8 (25%) | |
| Antiplatelet treatment at last follow-up, n % | 1/11 (9%) | 0 |
| Cytoreductive procedures at last follow-up, n (%) | 7/11 (64%) | 1/3 (33%) |
| Phlebotomy or erythrocytapheresis | 5/7 (71%) | 1/3 |
| Phlebotomy + HU | 2/7 (29%) | |
| Clinical outcome | (n = 11) | (n = 3) |
| Splenomegaly + reticulin fibrosis 2, n (%) | 2 (18%) | 0 |
| Time to evolution, mo | (38, 85) | |
| Median follow-up, mo (range) | 113 (24-210) | 28 (28-113) |
| Clinical and hematologic characteristics . | SP (n = 11) . | HP (n = 3) . |
|---|---|---|
| Male sex/female sex | 8/3 | 1/2 |
| Median age, y (range) | 16 (6-19) | 11 (11-14) |
| Median WBCs, × 109/L, (range) | 6.73 (4.86-28.67) | 6.76 (5.4-6.77) |
| Median Hct, % (range) | 53.9 (50-72.5) | 53.2 (42.4-54.5) |
| Median platelets, × 109/L, (range) | 217 (166-945) | 207 (202-271) |
| Symptoms, n (%) | 5/11 (45.5%) | 0 |
| Headache | 4/5 | |
| Paresthesias | 1/5 | |
| Splenomegaly, n (%) | 3/11 (27%) | 0/3 |
| Thrombotic events before/at diagnosis, % | 0 | 0 |
| Bleedings before/at diagnosis, % | 0 | 0 |
| Epo serum level, (range) | 4.5 (1.1-16.4) | 4.5; 5; 7.4 |
| BM biopsy, n (%) | 10/11 | 1/3 |
| Reticulin fibrosis 0-1/1-2, n (%) | 3/10 (30%) | 1/1 |
| Biologic markers | (n = 11) | (n = 3) |
| EEC positive, n (%) | 4/10 (40%) | 0/3 |
| JAK2V617, n (%) | 3/11 (27%) | 0/3 |
| V617F allele burden | 39%; 43.5%; 48% | |
| Clonal hemopoiesis, no. females, % | 2/3 | 0/2 |
| Treatment | (n = 11) | (n = 3) |
| Antiplatelet treatment, n (%) | 4/11 (36 %) | 0 |
| Cytoreductive therapy, n (%) | 8/11 (72%) | 1/3 (33%) |
| Phlebotomy or erythrocytapheresis | 6/8 (75%) | 1/3 |
| Phlebotomy + HU | 2/8 (25%) | |
| Antiplatelet treatment at last follow-up, n % | 1/11 (9%) | 0 |
| Cytoreductive procedures at last follow-up, n (%) | 7/11 (64%) | 1/3 (33%) |
| Phlebotomy or erythrocytapheresis | 5/7 (71%) | 1/3 |
| Phlebotomy + HU | 2/7 (29%) | |
| Clinical outcome | (n = 11) | (n = 3) |
| Splenomegaly + reticulin fibrosis 2, n (%) | 2 (18%) | 0 |
| Time to evolution, mo | (38, 85) | |
| Median follow-up, mo (range) | 113 (24-210) | 28 (28-113) |
SP indicates sporadic polycythemia; HP, hereditary polycythemia; n, number of patients; PV, polycythemia vera; and EEC, endogenous erythroid colony.