Trials · Topics · Bone Marrow Failure
Bone Marrow Failure clinical trials
11 Bone Marrow Failure trials (Malignant Hematology) summarized for fellows and board review, newest first within each disease. Each summary covers design, results, toxicity and what changed in practice.
Jump to: Aplastic Anemia · PNH · Telomere diseases / DKC
Aplastic Anemia (5)
- EMAA trial Blood 2026Adding eltrombopag to cyclosporine A as first-line therapy for moderate aplastic anemia with clinically significant cytopenia significantly improved trilineage...
- Eltrombopag + IST in Severe AA NEJM 2022Adding eltrombopag to standard IST improved the rate, rapidity, and strength of hematologic response vs IST alone in previously untreated SAA, without added toxicity.
- Townsley 2017 (SAA + eltrombopag) NEJM 2017Adding eltrombopag to standard hATG + cyclosporine substantially improved hematologic response rates in treatment-naïve SAA (94% vs ~66% historical), with a notably...
- Olnes 2012 (eltrombopag SAA) NEJM 2012Eltrombopag produced hematologic responses in 44% of patients with refractory SAA who had failed prior immunosuppression, establishing TPO receptor agonism as a novel...
- Scheinberg 2011 (Horse vs Rabbit ATG, SAA) NEJM 2011Rabbit ATG was unexpectedly inferior to horse ATG as first-line immunosuppressive therapy for severe aplastic anemia, with significantly lower response rates and...
PNH (5)
- COMMODORE 2 Am J Hematol 2024Crovalimab given primarily subcutaneously every 4 weeks was non-inferior to intravenous eculizumab every 2 weeks for LDH normalization in complement-naïve PNH, with a...
- PEGASUS NEJM 2021Pegcetacoplan significantly improved hemoglobin by +4.34 g/dL compared to eculizumab in patients with persistent anemia despite C5 inhibition, by addressing...
- ALXN1210-PNH-301/302 Blood 2019Ravulizumab q8w was non-inferior to eculizumab q2w across all efficacy endpoints in both complement-naive and eculizumab-switching PNH patients, offering equivalent...
- SHEPHERD Blood 2008Eculizumab substantially reduced hemolysis and improved fatigue in non-transfusion-dependent PNH patients with active hemolysis, confirming its benefit across the...
- TRIUMPH NEJM 2006Eculizumab eliminated transfusion requirement in 51% of patients and reduced hemolysis by 86%, establishing C5 inhibition as the standard of care for...
Telomere diseases / DKC (1)
- Townsley 2016 NEJM 2016Danazol 800 mg/day for 24 months led to telomere elongation and hematologic improvement in patients with telomere diseases including DKC and related bone marrow...