Ruxolitinib remains the standard frontline therapy for intermediate-2 and high-risk myelofibrosis, with phase 3 and real-world data showing spleen and symptom benefits. Cytopenias, transfusion needs and resistance after 2 to 3 years continue to drive interest in new combinations.
A phase 1 trial shows denileukin diftitox administered before CAR-T therapy has a favorable safety profile and encouraging efficacy in high-risk DLBCL patients, with an 86% overall response rate and 77% one-year progression-free survival. The treatment demonstrated consistent regulatory T-cell depletion, supporting its immunomodulatory mechanism. Larger controlled studies are needed to confirm these preliminary findings.
The myelofibrosis therapeutic pipeline now includes over 40 candidates from more than 35 companies, with recent developments including Orphan Drug Designation for CK0804 and multiple Phase III trial initiations. Key clinical milestones include completed enrollment in Karyopharm's SENTRY trial and upcoming Phase I data from Incyte's combination therapy studies.
The FDA approved Tecvayli plus Darzalex Faspro for relapsed/refractory multiple myeloma in March 2026, and Darzalex Faspro-based quadruplet D-VRd for newly diagnosed transplant-ineligible patients in January 2026.
AstraZeneca's CALQUENCE received FDA approval as the first all-oral, fixed-duration regimen for chronic lymphocytic leukemia, while the EMA validated the marketing authorization application for ENHERTU in HER2-positive breast cancer treatment.
CAR T-cell therapy is delivering high remission rates for multiple myeloma patients, with improved handoff communication reducing infection risks. FDA-approved BCMA-targeting products show response rates from 72% to 98% in heavily pretreated patients.
The FDA approved the combination of acalabrutinib and venetoclax as the first all-oral, fixed-duration regimen for adults with previously untreated chronic lymphocytic leukemia or small lymphocytic lymphoma, based on Phase 3 AMPLIFY trial results.
The FDA approved acalabrutinib plus venetoclax as the first all-oral, fixed-duration regimen for previously untreated chronic lymphocytic leukemia, based on phase III AMPLIFY trial results showing 35% reduction in disease progression or death risk versus chemoimmunotherapy.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT01295801 |
Vitamin B6 on Relieving Linezolid-associated Cytopenias |
UNKNOWN | |
| NCT01200017 |
Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant |
NO_LONGER_AVAILABLE | |
| NCT00002303 |
The Safety and Effectiveness of r-HuEPO in Patients With AIDS or Advanced AIDS-Related Complex (ARC) Who Are Anemic |
COMPLETED | NA |
| NCT00002302 |
The Safety and Effectiveness of r-HuEPO in Patients With AIDS and Anemia Caused by AIDS and Treatment With AZT |
COMPLETED | NA |
| NCT00002281 |
A Study of Granulocyte Colony-Stimulating Factor Plus Recombinant Erythropoietin Plus Zidovudine in Patients With AIDS or AIDS-Related Complex |
COMPLETED | NA |
| NCT00002263 |
A Pilot Study to Evaluate the Effects of Subcutaneously Administered Recombinant Human Granulocyte-Macrophage Colony Stimulating Factor in Pediatric HIV-Infected Patients With Neutropenia Secondary to Azidothymidine |
COMPLETED | NA |
| NCT00002258 |
A Phase I, Open Label Trial to Evaluate the Safety, Tolerance and Biological Effects of SDZ ILE-964 (Recombinant Human Interleukin-3, RhIL-3) in HIV Infected Patients With Cytopenia |
COMPLETED | PHASE1 |
| NCT00002126 |
A Randomized, Controlled, Multicenter Trial of Filgrastim (Recombinant-Methionyl Human Granulocyte Colony Stimulating Factor; G-CSF) for the Prevention of Grade 4 Neutropenia in Patients With HIV Infection. |
COMPLETED | PHASE2 |
| NCT00002073 |
A Double-Blind, Placebo-Controlled Study With Open-Label Follow-Up To Determine the Safety and Efficacy of Subcutaneous Doses of r-HuEPO in AIDS Patients With Anemia Induced by Their Disease and AZT Therapy |
COMPLETED | NA |
| NCT00002072 |
A Double-Blind, Placebo-Controlled Study With Open-Label Follow-Up To Determine the Safety and Efficacy of r-HuEPO in AIDS Patients With Anemia Induced by Their Disease and AZT Therapy |
COMPLETED | NA |
