This work is licensed under a Table 1.
Patient and treatment characteristics for participants vs non-participants and for patients who were prepubertal at transplant vs pubertal/postpubertal at transplant.
| Patient characteristics | Participants, n = 39 | Non-participants, n = 37 | Participants prepubertal at HSCT, n = 26 | Participants pubertal/postpubertal at HSCT, n = 13 |
|---|---|---|---|---|
| Age at HSCT, median (range) | 9.4 (0.4–16.9) | 9.7 (3.1–17.0) | 8.1 (0.4–13.8) | 15.1 (13.0–16.9) |
| Prepubertal stage at HSCT, n (%) | 26 (67) | NA | 26 (100) | 0 (0) |
| Age at last follow-up, median (range) | 27.6 (18.5–40.4) | 25.6 (18.4–38.5) | 26.1 (18.5–38.0) | 29.5 (24.8–40.4) |
| Years from HSCT at last follow-up, median (range) | 19.0 (7.7–27.3) | 15.4 (8.1–26.3) | 20.1 (7.7–27.3) | 15.8 (8.6–25.1) |
| Diagnosis (malignant vs non-malignant) | ||||
| Malignancies, n (%) | 29 (74) | 22 (59) | 19 (73) | 10 (77) |
| Acute lymphoblastic leukemia | 12 | 13 | 9 | 3 |
| Acute myeloid leukemia | 6 | 2 | 2 | 4 |
| Myelodysplastic syndrome | 5 | 2 | 5 | 0 |
| Non-Hodgkin lymphoma | 4 | 1 | 1 | 3 |
| Other leukemia (biphenotypic, JMML, CML) | 2 | 4 | 2 | 0 |
| Non-malignant diseases, n (%) | 10 (26) | 15 (41) | 7 (27) | 3 (23) |
| Severe aplastic anemia | 5 | 4 | 3 | 2 |
| Immune deficiency syndromes | 2 | 2 | 2 | 0 |
| Other non-malignant diseases | 3 | 9 | 2 | 1 |
| Donor match, n (%) | ||||
| Matched sibling donor | 14 (36) | 15 (41) | 9 (35) | 5 (38) |
| Matched unrelated donor | 19 (49) | 15 (41) | 12 (46) | 7 (54) |
| Alternative donor | 6 (15) | 7 (19) | 5 (19) | 1 (8) |
| Stem cell source, n (%) | ||||
| Bone marrow | 38 (97) | 33 (92) | 26 (100) | 12 (92) |
| Peripheral blood | 1 (3) | 4 (11)a | 0 | 1 (8) |
| Conditioning regimens | ||||
| BU + CY (+ MEL/VP16/THIO), n (%) | 14 (36) | 16 (43)b | 12 (46) | 2 (15) |
| TBI 2 Gy + CY, n (%) | 2 (5) | 0 | 1 (4) | 1 (8) |
| TBI 11.3–12 Gy + CY/BU-CY/VP16, n (%) | 23 (59) | 21 (57) | 13 (50) | 10 (77) |
| CNS shielding, n | 0 | 0 | 0 | 0 |
| Gonadal shielding, n | 2c | 1 | 1 | 1 |
| Additional irradiation at conditioning | ||||
| Testicular irradiation 4 Gy, n | 11 | 9 | 5 | 6 |
| CNS irradiation 10 Gy, n | 1 | 3 | 1 | 0 |
| Cumulative treatment (pre-HSCT plus conditioning) | ||||
| CED (g/m2), median (range) | 5.31 (0–28.74) | NA | 5.43 (0–23.46) | 4.48 (2.00–28.74) |
| Testicular irradiation (Gy), median (range) | 11.3 (0–36) | NA | 3.4 (0–36) | 12.0 (0–16) |
| CNS irradiation (Gy), median (range) | 12.0 (0–36) | NA | 11.3 (0–36) | 12.0 (0–12) |
| Treatment groups (cumulative treatment) | ||||
| Chemotherapy only, n | 13 | NA | 11 | 2 |
| Low-dose testicular irradiationd, n | 4 | NA | 2 | 2 |
| TBI 11.3–12 Gy, no add. irradiation, n | 7 | NA | 4 | 3 |
| TBI 11.3–12 Gy plus add. CNS irradiation, n | 3 | NA | 3 | 0 |
| TBI 11.3–12 Gy plus add. testis irradiation, n | 10 | NA | 4 | 6 |
| TBI 11.3–12 Gy plus add. testis and add. CNS irradiation, n | 2 | NA | 2 | 0 |
| Orchiectomy | ||||
| Unilateral, n | 4 | NA | 3 | 1 |
| Bilateral, n | 1 | NA | 1 | 0 |
| Graft vs host disease | ||||
| Acute graft vs host disease, n (%) | 23 (67) | 24 (65) | 18 (69) | 5 (38) |
| Grade I | 6 | 8 | 4 | 2 |
| Grade II | 13 | 14 | 10 | 3 |
| Grade III+IV | 4 | 2 | 4 | 0 |
aOne non-participant received both bone marrow and peripheral blood but was only counted in as receiving peripheral blood; bThree non-participants treated for severe aplastic anemia received conditioning with CY only, and one treated for Fanconi anemia received 4.5 Gy thoracoabdominal irradiation in addition to CY; cTesticular irradiation dose estimated to be 40% of the TBI dose; dLow-dose testicular irradiation included patients treated with TBI 2 Gy or TBI 11.3–12 Gy with gonadal shielding.
add., additional (to TBI); BU, busulfan; CED, cyclophosphamide equivalent dose; CML, chronic myeloid leukemia; CY, cyclophosphamide; HSCT, hematopoietic stem cell transplantation; JMML, juvenile myelomonocytic leukemia; MEL, melphalan; TBI, total body irradiation; THIO, thiotepa; VP16, etoposide.