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| Author | Study type | Drug | Mechanism of action | Treatment regimen | ORR | CR | Survival | Grade 3-4 AEs |
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| Jagasia et al. | Prospective, multicenter, open-label, single-cohort, phase II | Ruxolitinib | JAK1 and JAK2 kinase inhibitor | 5 mg BID with an increase to 10 mg BID | 73.2% | 56.3% | MS of 7.6 months | Thrombocytopenia (61%); anemia (45%); infections (40%); neutropenia (40%); hemorrhage (20%); fatigue (14%); elevated transaminases (14%); edema (13%); hypertension (13%); thrombosis (11%); dyspnea (7%); diarrhea (7%); headache (4%); and rash (3%) |
|
| Bordigoni et al. | Prospective, single-center, open-label, multicohort, phase II | Daclizumab | IL-2Rα antagonists | 1 mg/kg on days 1, 4, 8, 15, and 22 | 90.1% | 68.8% | 4-year EFS 54.6% | 38.7% CMV reactivation |
|
| Groth et al. | Prospective, single-arm, phase I/II for safety and efficacy | CD3/CD7-IT | Antibodies to CD3/CD7 antigens | Four doses 4 mg/m2 | 60% on day 28 | 50% | 6-months OS 60% | Grade 3: thrombocytopenia (15%), neutropenia (5%), elevated bilirubin (10%), myopathy (5%), microangiopathy (5%), hypoalbuminemia (5%) |
| 2-year OS 35% | Grade 4: thrombocytopenia (25%) |
|
| Socié et al. | Phase III, randomized, open-label, multicenter | Inolimomab | Monoclonal antibody to CD25 | Days 1–8 = 0.3 mg/kg/d IV for induction phase and 0.2 mg/kg/d IV for maintenance | NR | NR | 1-year OS 28.5% | At least one grade 3, 4, or 5 = 100% in treatment group; 98% in control group |
| If CR on day 9, maintenance injection on days 9–28 |
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| Mehta et al. | Prospective study | Vedolizumab | Monoclonal antibody which inhibits interaction between a4b7 on T-cells with MAdCAM-1 on endothelium | IV infusion at a dose of 300 mg at 0, 2, and 6 weeks, and then every 8 weeks depending on the response | 25% | 20% | 35% at 6 months | Infections, liver enzymes elevation |
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| Bacigalupo et al. | Analysis of 2 prospective studies | Begelomab | Monoclonal antibody to CD26 | From 2 to 4.5 mg/m2/day | 75% and 61% | 11% and 12% | 50% and 30% at 1 year | Group I: bronchopneumonia (25%), dyspnea (16.6%) |
| Group II: bacterial sepsis, convulsions, acute respiratory failure, multiorgan failure, E. coli infection with renal failure (0.06%, 1 patient) |
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| Bolaños-Meade et al. | Single-center, phase I dose-escalation study | Pentostatin | Nucleoside inhibitor | 1 to 4 mg/m2/d for 3 days | 76% | 63% | MS 85 days | Thrombocytopenia 4%, late infections 9% |
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| Benito et al. | Pilot trial for toxicity and efficacy | Sirolimus | mTOR inhibitor | 19% of patients 15 mg/m2 oral loading dose day 1 + 5 mg/m2/d for 13 days | 57% | 24% | 400-day OS 28% | Myelosuppression 9%, seizures 9% |
| 33% given 5 mg/m2/day for 14 days without loading dose |
| 48% received 4 mg/m2/day for 14 days without loading dose |
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| Carpenter et al. | Multicenter, single-arm, phase II | Visilizumab | Monoclonal antibody to CD25 and CD3 | First dose 3 mg/m2; second dose if recurrence in 2–6 weeks | 32% | 14% | 180-day OS 32% | Infusion reaction (4.5%) |
| 310-day OS 20.5% |
| MS 539 days |
|
| Ho et al. | Single-center, multi-cohort, prospective, phase I | Denileukin diftitox | Cytotoxicity against IL-2 receptors | 23% of patients 9 μg/kg on days 1 and 15 | 71% | 50% | 6.3–26.4 months OS 33% | Hepatic transaminitis (4%); infusion reaction (6%); acute renal failure (4%); cardiac tamponade (4%); pulmonary embolism (4%); sepsis (11%) |
| 60% of patients 9 μg/kg IV on days 1, 3, 5, 15, 17, and 19 (maximum tolerated dose) | MS 7.2 months |
| 17% of patients given 9 μg/kg IV days 1–5 and 15–19 |
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| Schroeder et al. | Open-label, parallel-cohort, multicenter, phase I | Itacitinib | JAK1-inhibitor | 200 mg once daily + corticosteroids or 300 mg once daily + corticosteroids | 75% in 200 mg group | 37.5% in 200 mg group | 6-month OS 58.6% | 200 mg group vs. 300 mg group: diarrhea (28.6% vs. 13.3%), edema (0% vs. 1%); abdominal pain (14.3% vs. 6.7%), hypokalemia (28.6% vs. 20.0%), hyperglycemia (21.4% vs. 26.7%), fatigue (28.6% vs. 0%), decreased appetite (14.3% vs. 6.7%), tachycardia (14.3% vs. 0%), hypoalbuminemia (21.4% vs. 6.7%), hypophosphatemia (21.4% vs. 13.3%), nausea (0% vs. 6.7%), and vomiting (0% vs. 6.7%) |
| 66.7% in 300 mg | 22.2% in 300 mg | 12-month OS 48.3% |
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| Marcondes et al. | Phase I/II open-label single-center study, | Alpha-1 antitripsin | Serine protease inhibitor with anti-inflammatory properties | Cohort 1: 90 mg/kg on day 1; 30 mg/kg/day on days 3, 5, 7, 9, 11, 13, and 15. Cohort 2: 90 mg/kg on day 1; 60 mg/kg/day | 66.6% | 33.3% | 50% at >104 to >820 days | None |
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