Patient pre-HSCT characteristics and HSCT and clinical variables
| Pt# . | Age at Dx . | Age at HSCT . | WBC (×109/L) . | Mono (K/μL) . | Plt (K/μL) . | HbF (%) . | PB blast (%) . | BM blast (%) . | Pre-HSCT chemo . | Cytogenetics mutation . | Sex (R/D) . | ABO (R/D) . | CMV (R/D) . | Regimen . | Graft type . | ANC >500 (×109/L) . | Plt >20 (K/μL) . | Donor chimerism (%) . | F/U (mo) . | Alive . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 0.6 | 0.8 | 60.0 | 16.8 | 89.0 | 18.0 | 5.0 | 8.0 | 6-MP* | 47,XX | F/F | A+/O+ | −/M+ | BuMel/ rATG | 4/6 UCB | 16 | 16 | 100 | 99.3 | Yes |
| Flu, Ara-C cis-R | Extra ring chromosome 3 NF1 | |||||||||||||||||||
| 2 | 4.0 | 4.2 | 30.4 | 2.4 | 37.0 | 52.0 | 3.0 | 9.0 | None | 46,XX | F/M | A+/O+ | −/− | BuMel/Flu | 9/10 MMSD | 17 | 93 | 100 | 83.9 | Yes |
| None | ||||||||||||||||||||
| 3 | 0.5 | 1.2 | 36.3 | 9.8 | 44.0 | 7.7 | 2.0 | 6.0 | 6-MP* | 46,XY | M/F | A+/O+ | −/M+ | BuMel/ rATG | 5/6 UCB | 27 | 133 | 96 | 41.9 | Yes |
| None | ||||||||||||||||||||
| 4 | 1.7 | 2.5 | 25.3 | 2.8 | 110.0 | 0.5 | 2.0 | 3.0 | 6-MP* | 46,XX | F/F | O+/O+ | +/− | BuMel | 10/10 MSD | 25 | 49 | 99 | 25.3 | Yes |
| None | ||||||||||||||||||||
| 5 | 0.8 | 3.8 | 130.0 | 27.3 | 43.0 | 4.8 | 4.0 | 5.0 | HU* | 46,XY | M/F | A+/A+ | +/+ | BuMel | 10/10 MSD | 15 | 18 | 100 | 9.1 | Yes |
| 6-MP | None | |||||||||||||||||||
| 6 | 1.4 | 2.2 | 20.0 | 1.1 | 19.0 | 7.6 | 2.0 | 3.0 | None | 46,XX PTPN11† | F/F | O+/O+ | −/M+ | BuMel/ rATG | 6/6 UCB | N/A | N/A | 6 | 7.9 | Yes |
| 7 | 2.5 | 3.2 | 38.9 | 2.0 | 22.0 | 42.2 | 6.0 | 3.0 | 6-MP* | 46,XY PTPN11† | M/M | B+/B+ | +/+ | BuMel/ Alem | 9/10 MUD | 22 | 23 | 100 | 6.0 | Yes |
| Pt# . | Age at Dx . | Age at HSCT . | WBC (×109/L) . | Mono (K/μL) . | Plt (K/μL) . | HbF (%) . | PB blast (%) . | BM blast (%) . | Pre-HSCT chemo . | Cytogenetics mutation . | Sex (R/D) . | ABO (R/D) . | CMV (R/D) . | Regimen . | Graft type . | ANC >500 (×109/L) . | Plt >20 (K/μL) . | Donor chimerism (%) . | F/U (mo) . | Alive . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 0.6 | 0.8 | 60.0 | 16.8 | 89.0 | 18.0 | 5.0 | 8.0 | 6-MP* | 47,XX | F/F | A+/O+ | −/M+ | BuMel/ rATG | 4/6 UCB | 16 | 16 | 100 | 99.3 | Yes |
| Flu, Ara-C cis-R | Extra ring chromosome 3 NF1 | |||||||||||||||||||
| 2 | 4.0 | 4.2 | 30.4 | 2.4 | 37.0 | 52.0 | 3.0 | 9.0 | None | 46,XX | F/M | A+/O+ | −/− | BuMel/Flu | 9/10 MMSD | 17 | 93 | 100 | 83.9 | Yes |
| None | ||||||||||||||||||||
| 3 | 0.5 | 1.2 | 36.3 | 9.8 | 44.0 | 7.7 | 2.0 | 6.0 | 6-MP* | 46,XY | M/F | A+/O+ | −/M+ | BuMel/ rATG | 5/6 UCB | 27 | 133 | 96 | 41.9 | Yes |
| None | ||||||||||||||||||||
| 4 | 1.7 | 2.5 | 25.3 | 2.8 | 110.0 | 0.5 | 2.0 | 3.0 | 6-MP* | 46,XX | F/F | O+/O+ | +/− | BuMel | 10/10 MSD | 25 | 49 | 99 | 25.3 | Yes |
| None | ||||||||||||||||||||
| 5 | 0.8 | 3.8 | 130.0 | 27.3 | 43.0 | 4.8 | 4.0 | 5.0 | HU* | 46,XY | M/F | A+/A+ | +/+ | BuMel | 10/10 MSD | 15 | 18 | 100 | 9.1 | Yes |
| 6-MP | None | |||||||||||||||||||
| 6 | 1.4 | 2.2 | 20.0 | 1.1 | 19.0 | 7.6 | 2.0 | 3.0 | None | 46,XX PTPN11† | F/F | O+/O+ | −/M+ | BuMel/ rATG | 6/6 UCB | N/A | N/A | 6 | 7.9 | Yes |
| 7 | 2.5 | 3.2 | 38.9 | 2.0 | 22.0 | 42.2 | 6.0 | 3.0 | 6-MP* | 46,XY PTPN11† | M/M | B+/B+ | +/+ | BuMel/ Alem | 9/10 MUD | 22 | 23 | 100 | 6.0 | Yes |
ABO, ABO blood group system; Alem, alemtuzumab; ANC, absolute neutrophil count; Ara-C, cytarabine; BM, bone marrow; BuMel, busulfan and melphalan; cis-R, cis-retinoic acid; CMV, cytomegalovirus; D, donor; Dx, diagnosis; F, female; Flu, fludarabine; F/U, follow-up; HbF, fetal hemoglobin; HU, hydroxyurea; M, male; MMSD, mismatched sibling donor; Mono, monocyte; MSD, matched sibling donor; MUD, matched unrelated donor; N/A, not applicable; PB, peripheral blood; Plt, platelet; Pt#, patient number; R, recipient; rATG, rabbit antithymocyte globulin; 6-MP, mercaptopurine; UCB, unrelated cord blood; WBC, white blood cell count.
Pre-HSCT chemotherapy was administered to patients who had progressive/high WBC (≥35K), pulmonary/respiratory compromise, and/or prominent/progressive organomegaly before proceeding to HSCT.
Somatic mutation.