Experts at The Johns Hopkins Kimmel Cancer are nationally and internationally recognized as leaders in the research and treatment of paroxysmal nocturnal hemoglobinuria and other blood disorders. Our experienced and dedicated team provides our patients with innovative treatments and the best quality of care possible.

Research Papers

Characterization of breakthrough hemolysis events observed in the phase 3 randomized studies of ravulizumab versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria. Haematologica. 2021 Jan 1;106(1):230-237. doi: 10.3324/haematol.2019.236877. PMID 31949012

Summary:
Although eculizumab is the first-line treatment for paroxysmal nocturnal hemoglobinuria, between 11% to 27% of patients may still have some incidents of red blood cell destruction (hemolysis) on approved doses of the drug. In clinical trials, Robert Brodsky and colleagues found that a new PNH drug called ravulizumab, which lasts longer in the body and blocks the same C5 protein as eculizumab, can reduce the risk of breakthrough hemolysis in PNH patients by eliminating breakthrough cases caused by less-than-optimal blockage of C5 by eculizumab.

Eculizumab Bridging before Bone Marrow Transplant for Marrow Failure Disorders Is Safe and Does Not Limit Engraftment. Biol Blood Marrow Transplant. 2018 Dec;24(12):e26-e30. doi: 10.1016/j.bbmt.2018.07.032. Epub 2018 Jul 26. PMID 30055352.

Summary:
This study by Amy DeZern, Robert Brodsky and Richard Jones explores whether eculizumab affects the success of bone marrow transplant in patients with severe aplastic anemia and paroxysmal nocturnal hemoglobinuria. Eight patients with these disorders were treated with eculizumab and then proceeded to transplant. All were successfully transplanted and cured of their respective disorders. The researchers conclude that eculizumab is safe and effective in patients with PNH who require transplant, even if the marrow donors are only half-matched or fully matched but not related to the patient.

How I treat paroxysmal nocturnal hemoglobinuria. Blood. 2021 Mar 11;137(10):1304-1309. doi: 10.1182/blood.2019003812. PMID 33512400.

Summary:
In this paper, Robert Brodsky discusses the current state of treatment for paroxysmal nocturnal hemoglobinuria. Drugs such as eculizumab are very highly effective for treating red blood cell destruction and lowering the risk of blood clots, he notes, but do not treat bone marrow failure. He compares the uses and limitations of the two FDA approved drugs eculizumab and ravulizumab, as well as new drugs that are in development. He also discusses some rare indicators for a bone marrow transplant in PNH patients.