Table 1.
Summary of publications describing use of ruxolitinib in patients with HLH.
| Reference | N | Mean age (range) | HLH type | Therapy (N) | Ruxolitinib dose | Ruxolitinib duration | Concurrent HLH treatment (N) | ORR (CR, PR) | Overall survival† |
|---|---|---|---|---|---|---|---|---|---|
| Acosta et al. (43) | 1 | 35 y | Secondary (EBV/CMV, HIV) | Frontline | 15 mg BID | 4 weeks | Dex | 100% (CR) | 100% (4 weeks) |
| Ahmed et al. (44) | 7 | 41 y (29–62) | Secondary (1 AOS, 1 CMV, 1 SLE, 4 unknown) | Frontline (5) Rel/Ref (2) |
15 mg BID | 14–28 days | None (2) Steroids (5) | 100% (43%, 57%) | 100% (60 days) |
| Broglie et al. (45) | 1 | 11 y | Secondary (unknown) | Rel/Ref | 2.5 mg BID | NR | Dex & Anakinra | 100% | 100% (30 days) |
| Fuchs et al. (46) | 1 | 33 y | Secondary (Falciparum malaria) | Rel/Ref | 5 mg BID | 6 weeks | Dex & Etoposide | 100% | 100% (3 months) |
| Goldsmith et al. (47) | 2 | 25 y (24–26) | Secondary (1 EBV, 1 SJIA) | Rel/Ref | 5–20 mg BID | 3 months (1) NR (1) |
None (1) Alemtuzumab & steroids (1) | 100% (CR) | 100% (3 months) |
| Jianguo et al. (48) | 1 | 6 y | Secondary (PEG-IFNα-2a for chronic HBV) | Frontline | 2.5 mg BID | 3 months | None | 100% (CR) | 100% (1 year) |
| Levy et al. (49) | 1 | 16 y | Secondary (SPTCL, TIM3 def.) | Rel/Ref | 15 mg BID 20 mg BID | 2 months 10 months |
Cyclosporine & Anakinra | 100% (CR) | 100% (19 months) |
| Ramanan et al. (50) | 1 | 3 y | Primary (UNC13D) | Rel/Ref | 2.5 mg BID | 4 weeks | Steroids | 100% (CR) | 100% (4 weeks) |
| Sin et al. (51) | 1 | 38 y | Secondary (EBV) | Rel/Ref | 20 mg BID (10 mg BID w/ renal failure) | 7 days | None | 100% (PR) | 0% (7 days) |
| Slostad et al. (52) | 1 | 71 y | Secondary (Histoplasmosis, RA) | Frontline | 10 mg BID | 8 days +3 wk taper |
Steroids | 100% | 100% (3 weeks) |
| Trantham et al. (53) | 2 | 45 y (24–66) | Secondary (lymphoma) | Rel/Ref | 10–15 mg BID | NR | None (1) Alemtuzumab & anakinra (1) | 100% (CR1§) | 0% (8 months) |
| J Wang et al. (54) | 34 | 25.5 y (2–70) | Primary (1 PRF1) Secondary (25 EBV, 2 MAS, 6 unknown) | Rel/Ref | 0.3 mg/kg/day 5 mg BID (<14 y, ≥25 kg) 2.5 mg BID (<25 kg) | NR | None (16) Steroids (18) | 73.5% (14.7% 58.8%) | 56% (4–52 weeks) |
| H Wang et al. (55) | 3 | 40 y (24–52) | Secondary (2 lymphoma, 1 AOS) | Frontline | 5 mg BID | 4 weeks +1 wk QD |
Dex & Etoposide | 100% (33% CR, 66% PR) | 33% |
| Wei et al. (56) | 9 | 1.7 y (9m−5y) | Primary (2 PRF1) Secondary (5 EBV, 1 autoinflammatory disease, 1 unknown) | Rel/Ref | 2.5 mg BID (<10 kg) 5 mg BID (10–25 kg) 10 mg BID (>25 kg) | 1–4 weeks | NR | 33% (33% CR, 0% PR) | 89% (10–15 months*) |
| Zandvakili et al. (57) | 1 | 72 y | Secondary (Histoplasmosis, IBD, RA) | Frontline | 10 mg BID | 5 days +3 wk taper |
None | 100% (CR) | 100% (6 weeks) |
| Zhang et al. (58) | 12 | 4.7 y (1–13) | Secondary (8 EBV, 2 autoinflammatory disorder, 2 unknown) | Frontline | 2.5 mg BID (≤10 kg) 5 mg BID (11–20 kg) 10 mg BID (>20 kg) | 3–28 days | None | 83.3% (66.7% CR, 8.3% PR) | 91.7% (10 months**) |
| Zhao et al. (59) | 1 | 14 y | Primary (RAB27A) | Rel/Ref | 5–25 mg BID | 2 months | Steroids | 100% | 100% (8 months) |
| Zhou et al. (60) | 36 | 44.7 y (31–58) | Secondary (lymphoma) | Frontline | 0.3 mg/kg/day | 14 days | Dex, Etoposide, Doxorubicin | 83.3% (27.8% CR, 55.6% PR) | 40% (5 months) |
AOS, Adult-onset Stills disease; BID, twice daily; CMV, Cytomegalovirus; CR, complete response; Dex, Dexamethasone; EBV, Epstein-Barr virus; HBV, Hepatitis B virus; IBD, Inflammatory bowel disease; NR, Not reported; PR, partial response; QD, once daily; RA, Rheumatoid arthritis; Rel/Ref, Relapsed/Refractory; SJIA, Systemic juvenile idiopathic arthritis; SLE, Systemic lupus erythematosus; SPTCL, Subcutaneous panniculitis-like T-cell lymphoma.
Overall survival reported as percentage patients alive at publication, with time from initiation of ruxolitinib to end point (last follow-up or death) in parentheses when available.
Survival reported only for patients who completed full 4 weeks ruxolitinib since therapy was switched if no CR by 1 week.
Survival reported only for patients who received ruxolitinib alone and did not go on to receive HLH-94 therapy.
Both patients achieved CR1, but both subsequently relapsed (one while on ruxolitinib taper, one after 24 days ruxolitinib).