|
| Author | Study type | Drug | Mechanism of action | Treatment regimen | ORR | CR | Survival | Grade 3-4 AEs |
|
| Zeiser et al. | Multicenter, randomize, open-label phase 3 trial | Ruxolitinib | JAK1 and JAK2 kinase inhibitor | 10 mg BID | 40% | NR | NR | Thrombocytopenia (33%) |
| Anemia (30%) |
| CMV infection (26%) |
|
| Carpenter et al. | Open-label, multicenter, international phase 1/2 study | Ibrutinib | Bruton tyrosine kinase inhibitor | <12 years: Once daily from 120 mg/m2 to 240 mg/m2; patients older than 12 years received 420 mg | 77% in R/R group | 4% in R/R group | 18-months OS 91% in R/R group | 64% overall; pyrexia (8%), |
| Hypoxia (7%), |
| Neutropenia (7%), |
| Osteonecrosis (7%), |
| Stomatitis (7%), |
| ALT elevation (5%), |
| Hypokalemia (5%), |
| Pneumothorax (5%) |
|
| Cutler et al. | Phase 2 randomized multicenter registration trial | Belumosudil | ROCK2 inhibitor | 200 mg daily or 200 mg BID | ORR 74% in 200 mg daily group; 77% in 200 mg twice daily group | 6% in 200 mg daily group; 3% in 200 mg twice daily group | 89% in 2 years | 54% overall; pneumonia (8%), hypertension (6%), hyperglycemia (5%), anemia (3%), neutropenia (2%) |
| Noriko et al. | Multicenter, open label, single-arm study | Ibrutinib | Bruton tyrosine kinase inhibitor | 420 mg once daily or 280 mg if concomitant voriconazole | 73.7% | 10.5% | Not reached | 78.9% overall; |
| Pneumonia 21.1% |
| Cellulitis 15.8% |
| Stomatitis 10.5% |
| Decreased platelet count 10.5% |
|
| Chen et al. | Phase 1/2 clinical trial | Nilotinib | Tyrosine kinase inhibitor | From 200 mg to 400 mg twice daily up to the maximum tolerated dose | 46% | 0% | NR | Aspiration pneumonia (3%) |
| Hyponatremia (3%) |
| Hypophosphatemia (10%) |
| Influenza B (3%) |
| Joint stiffness (3%) |
| Lipase elevation (7%) |
| LFTs elevation (3%) |
| Muscle cramping (3%) |
| Myocardial infarction (3%) |
| Neutropenia (3%) |
| Shingles (3%) |
| Pancreatitis (3%) |
| P. jiroveci pneumonia (3%) |
| URI (3%) |
|
| Kitko et al. | Phase I/II open-label study | Axatilimab | Monoclonal antibody to CSF-1R | 3 mg/kg every four week | 82% | NR | 12 months FFS was 77% | 20% overall; |
| Hypertension (4%), |
| CPK increase (4%), |
| Pneumonia (3%), |
| AKI (2%), |
| AST increase (2%), |
| GGT increase (2%), |
| Lipase increase (2%), |
| Fever (2%) |
|
| DeFilipp et al. | Phase 1 clinical trial | Sonidegib | Hedgehog signaling pathway inhibitor | Up to 12 cycles of 28 days: 200 mg/day (dose level 1), 400 mg/day (dose level 2), and 600 mg/day (dose level 3) | 47% | 0% | NR | 100% overall; |
| Arthralgia (18%), |
| Abdominal pain (18%) |
| Myalgia (18%) |
| Back pain (12%) |
| Headache (12%) |
| Hypercalcemia (12%) |
| Single events (6%): anemia, cardiac arrest, chest pain, congestive heart failure, diarrhea, hypertension, hypotension, port infection, skin discoloration, small bowel obstruction, and superior vena cava syndrome |
|
| Cutler et al. | Open label phase 1/2 trial | Rituximab | Monoclonal antibody to CD20 | 38 cycles: 375 mg/m2 per week for 4 weeks | 70% | 10% | NR | 43% overall |
| Infectious diarrhea (14%) |
| Viral conjunctivitis (5%) |
| Hepatitis B reactivation (5%) |
| Septic arthritis (5%) |
| GI hemorrhage (5%) |
| Acute infusion reaction (5%) |
|
| Whangbo et al. | Single center phase 1 clinical trial | IL-2 | Enhancement of regulatory T-cell expansion | Children: 0.33 × 106 IU/m2; adults: 0.67 × 106 IU/m2 daily | 82% in pediatric patients | 0% | In 12 months | Pediatric AE: hypophosphatemia (9%) |
| Adults: 28.5% | Pediatric patients: 91% | Hypokalemia (9%) |
| Adults: 90% | Adult AE: |
| Transaminitis (20%) |
| Infection (10%) |
|
| Curtis et al. | Randomized phase 2 trial | Pomalidomide | Immune modulator | 0.5 mg per day orally or 2 mg per day; maximum dose was 2 mg per day | 47% | 0% | NR | Lymphocytopenia (28%) |
| Pneumonia (19%) |
| Hypophosphatemia (15%) |
| Fatigue (19%) |
| Maculopapular rash (3%) |
| Skin infection (3%) |
| Anemia (6%) |
| Limb edema (3%) |
| Hypotension (9%) |
| Increased ALT (6%) |
| Diarrhea (6%) |
| Hypertension (3%) |
| Leukocytopenia (3%) |
|
