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. 2009 Jan;46(1 Suppl 1):S1-S16.
doi: 10.1053/j.seminhematol.2008.11.004.

The management of paroxysmal nocturnal hemoglobinuria: recent advances in diagnosis and treatment and new hope for patients

Neal S Young  1 , Gabrielle Meyers, Hubert Schrezenmeier, Peter Hillmen, Anita Hill
Affiliations

The management of paroxysmal nocturnal hemoglobinuria: recent advances in diagnosis and treatment and new hope for patients

Neal S Young et al. Semin Hematol. 2009 Jan.

Abstract

PNH is a rare clonal hematopoietic stem disorder clinically characterized by the triad of chronic complement-mediated hemolysis, thrombosis, and bone marrow failure. While median survival has improved when historical data are compared to more recent data, thrombosis, the major cause of death in PNH, is still observed in approximately 40% of patients. The symptoms associated with this disorder–including fatigue, pain, esophageal spasm, and erectile dysfunction–are often severe and disabling. While PNH may be a curiosity to the physician, it forces the majority of patients to significantly modify their lives. Transplantation represents a curative option; however, the risks associated with this option are not insignificant. Eculizumab has been shown to significantly reduce hemolysis, improve anemia, reduce transfusion requirements, and significantly improve fatigue and other QoL scores. Clearly, targeted complement inhibition by eculizumab has the promise to significantly improve the lives of patients with PNH.

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Figures

Figure 1
Figure 1
Paroxysmal Nocturnal Hemoglobinuria (PNH): A Triad of Clinical Features
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Figure 2
Pathogenesis of PNH
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Figure 3
Eculizumab and Hemolysis
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Figure 4
Transfusion Requirement
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Figure 5
Apoptosis and FAS Expression in CD34 Cells Derived From Patients With PNH
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Figure 6
Site of Thrombosis in PNH
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Figure 7
The Ham Test
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Figure 8
Flow Cytometry Testing in PNH
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Figure 9
High Resolution Melting Analysis
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Figure 11
PNH Treatment Options
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Figure 12
Allogeneic Stem Cell Transplantation: Overall Survival (Data From the Working Party on Aplastic Anemia of the European Group for Blood and Marrow Transplantation (EBMT)
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Figure 13
Complement Cascade and Eculizumab
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Figure 14
Eculizumab PNH Clinical Program
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Figure 15
Levels of LDH During Eculizumab Treatment
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Figure 16
Complications in 467 Patients With PNH
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Figure 17
Effect of PNH Clone Size on Thrombosis
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Figure 18
Thrombosis: Overall Analysis
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Figure 19
Impact of PNH in Patients
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Strategies to Improve QoL
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Figure 21
TRIUMPH: Improvement in Fatigue (FACIT-F) With Eculizumab
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Figure 22
TRIUMPH Study: EORTC QLQ-C30
See this image and copyright information in PMC

References

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