New drugs for R/R wAIHA
| Treatment . | Dose schedule . | Response rate, % . | Comments . | Ref. . |
|---|---|---|---|---|
| Parsaclisib (PI3Ki) | 1 to 2.5 mg/d | n/a | Open-label phase 2 study | 72 |
| Pegcetacoplan (C3i) | 270 mg-360 mg/d | 40-50 | Open-label phase 2 study | 73 |
| Orilanolimab (FcRni) | n/a | n/a | Open-label phase 1b/2 study: phase 2 randomized, double-blind, placebo-controlled study | 74,75 |
| Nipocalimab (FcRni) | IV infusion every 2 or 4 wk | n/a | Phase 2 randomized, double-blind, placebo-controlled study | 76 |
| Fostamatinib (SyKi) | 100 to 150 mg, twice daily | 44 (time to response 2-30 wk) | Open-label phase 2 study; phase 3 randomized, double-blind, placebo-controlled study | 77,78 |
| Bortezomib (proteasome-i) | 1.3 mg/m2 subcutaneous ×2/wk for 2 wk | n/a | Open-label phase 2 study in association with anti-CD20 MoAb | 79 |
| Interleukin-2 | Cycle 1: 1.5 million IU per day for 9 wk; further cycles: 3 million IU per day for 16 wk | n/a | Open-label phase 1/2 study | 80 |
| Daratumumab (anti-CD38 MoAb) | 16 mg/kg week IV | n/a | Case reports in AIHA secondary to BM transplant | 81,82 |
| Sirolimus (mTORi) | 2 mg/m2 (adjusted to achieve a steady state of 5–10 ng/mL) | 100 | Cases reports of AIHA secondary to combined liver, small bowel, and pancreas transplant | 83 |
| Abatacept (CTLA-4 Ig) | 10 mg/kg monthly | 100 | Case reports in AIHA secondary to BM transplant | 84 |
| Imlifidase (ADCCi) | n/a | n/a | Studied in IgG-mediated diseases and transplant rejection | 85,86 |
| Treatment . | Dose schedule . | Response rate, % . | Comments . | Ref. . |
|---|---|---|---|---|
| Parsaclisib (PI3Ki) | 1 to 2.5 mg/d | n/a | Open-label phase 2 study | 72 |
| Pegcetacoplan (C3i) | 270 mg-360 mg/d | 40-50 | Open-label phase 2 study | 73 |
| Orilanolimab (FcRni) | n/a | n/a | Open-label phase 1b/2 study: phase 2 randomized, double-blind, placebo-controlled study | 74,75 |
| Nipocalimab (FcRni) | IV infusion every 2 or 4 wk | n/a | Phase 2 randomized, double-blind, placebo-controlled study | 76 |
| Fostamatinib (SyKi) | 100 to 150 mg, twice daily | 44 (time to response 2-30 wk) | Open-label phase 2 study; phase 3 randomized, double-blind, placebo-controlled study | 77,78 |
| Bortezomib (proteasome-i) | 1.3 mg/m2 subcutaneous ×2/wk for 2 wk | n/a | Open-label phase 2 study in association with anti-CD20 MoAb | 79 |
| Interleukin-2 | Cycle 1: 1.5 million IU per day for 9 wk; further cycles: 3 million IU per day for 16 wk | n/a | Open-label phase 1/2 study | 80 |
| Daratumumab (anti-CD38 MoAb) | 16 mg/kg week IV | n/a | Case reports in AIHA secondary to BM transplant | 81,82 |
| Sirolimus (mTORi) | 2 mg/m2 (adjusted to achieve a steady state of 5–10 ng/mL) | 100 | Cases reports of AIHA secondary to combined liver, small bowel, and pancreas transplant | 83 |
| Abatacept (CTLA-4 Ig) | 10 mg/kg monthly | 100 | Case reports in AIHA secondary to BM transplant | 84 |
| Imlifidase (ADCCi) | n/a | n/a | Studied in IgG-mediated diseases and transplant rejection | 85,86 |
ADCCi, ADCC inhibitor; C3i, complement component C3 inhibitor; CTLA-4 Ig, fusion protein of CTLA-4 with the Fc region of IgG; FcRni, neonatal crystallizable fragment receptor inhibitor; MoAb, monoclonal antibody; mTORi, mammalian target of rapamycin inhibitor; PI3Ki, phosphatidylinositol-4,5-bisphosphate 3-kinase δ type inhibitor; Proteasome-1, proteasome inhibitor; Syki, spleen tyrosine kinase inhibitor.