Prospective studies of pre-HCT ruxolitinib therapy
| Reference . | Number of patients recruited . | Number of patients who underwent HCT . | Splenectomy prior to HCT, n (%) . | Taper . | Ruxolitinib discontinuation . | SAE during ruxolitinib taper . | Conditioning . | OS at 2 y, % . | NRM at 2 y, % . | GF, % . | GVHD . |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Gupta et al. (2019)47 | 21 | 19* 5 MSD 14 UD | 0 (0) | 4 days | 1 day prior to start of conditioning | None | RIC (Flu-Bu) | 63 | 28% | 16 | aGVHD grade 3-4, 16% cGVHD 76% at 2 y |
| Salit et al. (2020)48 | 34 | 28† 14 MRD 11 UD 3 UCB | 3 (11) | 5 mg every 3 days | After 2-3 days of conditioning therapy | None | Multiple | 86 | 7% | 0 | aGVHD grade 3-4, 22% cGVHD, 41% |
| Robin et al. (2021)46 | 64 | 59‡ 18 MSD 32 MUD 14 UD 9/10 | 19 (30) | 15 days (n = 17) | 1 day prior to start of conditioning | TLS 3§ RWS 3 Heart failure | RIC (Flu-Mel) | 55 | 46% 23% MSD 50% MUD 77% UD 9/10 | 0 | aGVHD grade 3-4, 44% cGVHD 37% at 2 y |
| None (n = 42) | 1 day prior to start of conditioning | Heart failure |
| Reference . | Number of patients recruited . | Number of patients who underwent HCT . | Splenectomy prior to HCT, n (%) . | Taper . | Ruxolitinib discontinuation . | SAE during ruxolitinib taper . | Conditioning . | OS at 2 y, % . | NRM at 2 y, % . | GF, % . | GVHD . |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Gupta et al. (2019)47 | 21 | 19* 5 MSD 14 UD | 0 (0) | 4 days | 1 day prior to start of conditioning | None | RIC (Flu-Bu) | 63 | 28% | 16 | aGVHD grade 3-4, 16% cGVHD 76% at 2 y |
| Salit et al. (2020)48 | 34 | 28† 14 MRD 11 UD 3 UCB | 3 (11) | 5 mg every 3 days | After 2-3 days of conditioning therapy | None | Multiple | 86 | 7% | 0 | aGVHD grade 3-4, 22% cGVHD, 41% |
| Robin et al. (2021)46 | 64 | 59‡ 18 MSD 32 MUD 14 UD 9/10 | 19 (30) | 15 days (n = 17) | 1 day prior to start of conditioning | TLS 3§ RWS 3 Heart failure | RIC (Flu-Mel) | 55 | 46% 23% MSD 50% MUD 77% UD 9/10 | 0 | aGVHD grade 3-4, 44% cGVHD 37% at 2 y |
| None (n = 42) | 1 day prior to start of conditioning | Heart failure |
One patient with disease progression to AML, 1 patient with sudden death during ruxolitinib phase.
Three patients with progression to AML during ruxolitinib phase, 3 patients not meeting criteria for transplant.
One donor withdrawal. One patient continued taking ruxolitinib, 3 patients died without HCT; 2 with heart failure following splenectomy.
No TLS following protocol amendment to abrupt ruxolitinib discontinuation.
aGVHD, acute GVHD; AML, acute meloid leukemia; cGVHD, chronic GVHD; Flu-Bu, fludarabine/busulfan; Flu-Mel, fludarabine-melphalan; GF, graft failure; RIC, reduced-intensity conditioning; RWS, ruxolitinib withdrawal syndrome; SAE, significant adverse event; TLS, tumor lysis syndrome; UCB, umbilical cord blood.