Paroxysmal Nocturnal Hemoglobinuria: Current Management, Unmet Needs, and Recommendations
Monika Oliver, Christopher J Patriquin · 2023
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Description
Monika Oliver and Christopher Patriquin reviewed care for a rare blood disease in 2023, setting out what works now and what is still missing. In this disease, the body's own defense system destroys red blood cells, causing anemia, blood clots, and organ strain. With basic care alone, about a third of patients die.
New drugs that block parts of the defense system have changed the outlook, raising blood counts and cutting clots and transfusions. Longer-acting and oral options now exist, and some help patients who stay anemic on older drugs. Gaps remain, such as care in pregnancy and guarding against infection. The authors back a shared world registry to track how the new drugs perform over time.
Catalog record
| CREATOR | Monika Oliver, Christopher J Patriquin |
|---|---|
| YEAR | 2023 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 49 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |