What is a potential curative treatment for Paroxysmal Nocturnal Hemoglobinuria?

What is a potential curative treatment for Paroxysmal Nocturnal Hemoglobinuria?

• A. Immunoglobulin therapy
• B. Iron chelation
• C. Bone marrow transplant
• D. Bone marrow biopsy not curative

Answer: (C)

Paroxysmal nocturnal hemoglobinuria is a clonal disorder of hematopoietic stem cells that lacks GPI-anchored proteins, leading to complement-mediated red cell destruction. The way to potentially cure it is to replace the diseased marrow with healthy donor marrow through an allogeneic bone marrow transplant. By repopulating the hematopoietic system with donor stem cells that produce normal GPI-anchored proteins, the abnormal PNH clone can be eliminated, curing the underlying problem. Treatments that reduce hemolysis, like complement inhibitors, help symptoms and reduce complications but do not eradicate the clone, so they’re not curative. Immunoglobulin therapy or iron chelation address other issues or complications but don’t cure PNH. A bone marrow biopsy is a diagnostic procedure, not a treatment.

Paroxysmal nocturnal hemoglobinuria is a clonal disorder of hematopoietic stem cells that lacks GPI-anchored proteins, leading to complement-mediated red cell destruction. The way to potentially cure it is to replace the diseased marrow with healthy donor marrow through an allogeneic bone marrow transplant. By repopulating the hematopoietic system with donor stem cells that produce normal GPI-anchored proteins, the abnormal PNH clone can be eliminated, curing the underlying problem.

Treatments that reduce hemolysis, like complement inhibitors, help symptoms and reduce complications but do not eradicate the clone, so they’re not curative. Immunoglobulin therapy or iron chelation address other issues or complications but don’t cure PNH. A bone marrow biopsy is a diagnostic procedure, not a treatment.