A patient with nocturnal paroxysmal hemoglobinuria in everyday medical practice: from a primary care physician to a hematologist – a case report Case report
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Abstract
Nocturnal paroxysmal hemoglobinuria is an acquired hemolytic anemia characterized by a triad of symptoms: intravascular hemolysis, pancytopenia, and a tendency to venous thrombosis. In patients with nocturnal paroxysmal hemoglobinuria, these symptoms occur in various combinations. The occurrence of several attacks of intravascular hemolysis results in episodes of thrombosis at unusual sites, and these patients may have varying degrees of bone marrow dysfunction. Diagnosis can be confirmed by blood granulocyte flow cytometry using FLAER tests. The treatment strategy of complement inhibition with eculizumab, although very expensive, is very effective.
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How to Cite
Kozińska, J. (2023). A patient with nocturnal paroxysmal hemoglobinuria in everyday medical practice: from a primary care physician to a hematologist – a case report. Medycyna Faktow (J EBM), 16(2(59), 255-260. https://doi.org/10.24292/01.MF.0223.20
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References
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3. Hill A, Kelly R, Hillmen P. Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood. 2013; 121(25): 4985-96. http://doi.org/10.1182/blood-2012-09-311381.
4. Luzzatto L, Gianfaldoni G, Notaro R. Management of paroxysmal nocturnal haemoglobinuria: a personal view. Br J Haematol. 2011; 153(6): 709-20. http://doi.org/10.1111/j.1365-2141.2011.08690.x.
5. Bektas M, Copley-Merriman C, Khan S et al. Paroxysmal nocturnal hemoglobinuria: current treatments and unmet needs. J Manag Care Spec Pharm. 2020; 26(12-b Suppl): S14-S20. http://doi.org/10.18553/jmcp.2020.26.12-b.s14.
6. ChPL Eculizumab strona internetowa.
7. Dmytrijuk A, Robie-Suh K, Cohen MH et al. FDA report: eculizumab (Soliris) for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Oncologist. 2008; 13(9): 993-1000. http://doi.org/10.1634/theoncologist.2008-0086.
8. Szlendak U, Budziszewska B, Spychalska J et al. Paroxysmal nocturnal hemoglobinuria: advances in the understanding of pathophysiology, diagnosis, and treatment. Pol Arch Intern Med. 2022; 132(6): 16271.
9. Bohl S, Kuchenbauer S, von Harsdorf S et al. Thrombotic Microangiopathy after Allogeneic Stem Cell Transplantation: A Comparison of Eculizumab Therapy and Conventional Therapy. Biol Blood Marrow Transplant. 2017; 23: 2172-7.
10. Brodsky RA. How I treat paroxysmal nocturnal hemoglobinuria. Blood. 2021; 137(10): 1304-9. http://doi.org/10.1182/blood.2019003812.
11. Aktualności. Portal hematoonkologia.pl 03.03.2023. Czy będzie nowy program lekowy dla chorych na nocną napadową hemoglobinurię?
12. Ter Avest M, Bouwmeester RN, Duineveld C et al.; CUREiHUS study group. Proposal for individualized dosing of eculizumab in atypical haemolytic uraemic syndrome: patient friendly and cost-effective. Nephrol Dial Transplant. 2023; 38(2): 362-71. http://doi.org/10.1093/ndt/gfac056.
13. Loschi M, Porcher R, Barraco F et al. Impact of eculizumab treatment on paroxysmal nocturnal hemoglobinuria: a treatment versus no-treatment study. Am J Hematol. 2016; 91(4): 366-70. http://doi.org/10.1002/ajh.24278.
2. de Latour RP, Mary JY, Salanoubat C et al. French Society of Hematology, French Association of Young Hematologists. Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories. Blood. 2008; 112(8): 3099-106. http://doi.org/10.1182/blood-2008-01-133918.
3. Hill A, Kelly R, Hillmen P. Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood. 2013; 121(25): 4985-96. http://doi.org/10.1182/blood-2012-09-311381.
4. Luzzatto L, Gianfaldoni G, Notaro R. Management of paroxysmal nocturnal haemoglobinuria: a personal view. Br J Haematol. 2011; 153(6): 709-20. http://doi.org/10.1111/j.1365-2141.2011.08690.x.
5. Bektas M, Copley-Merriman C, Khan S et al. Paroxysmal nocturnal hemoglobinuria: current treatments and unmet needs. J Manag Care Spec Pharm. 2020; 26(12-b Suppl): S14-S20. http://doi.org/10.18553/jmcp.2020.26.12-b.s14.
6. ChPL Eculizumab strona internetowa.
7. Dmytrijuk A, Robie-Suh K, Cohen MH et al. FDA report: eculizumab (Soliris) for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Oncologist. 2008; 13(9): 993-1000. http://doi.org/10.1634/theoncologist.2008-0086.
8. Szlendak U, Budziszewska B, Spychalska J et al. Paroxysmal nocturnal hemoglobinuria: advances in the understanding of pathophysiology, diagnosis, and treatment. Pol Arch Intern Med. 2022; 132(6): 16271.
9. Bohl S, Kuchenbauer S, von Harsdorf S et al. Thrombotic Microangiopathy after Allogeneic Stem Cell Transplantation: A Comparison of Eculizumab Therapy and Conventional Therapy. Biol Blood Marrow Transplant. 2017; 23: 2172-7.
10. Brodsky RA. How I treat paroxysmal nocturnal hemoglobinuria. Blood. 2021; 137(10): 1304-9. http://doi.org/10.1182/blood.2019003812.
11. Aktualności. Portal hematoonkologia.pl 03.03.2023. Czy będzie nowy program lekowy dla chorych na nocną napadową hemoglobinurię?
12. Ter Avest M, Bouwmeester RN, Duineveld C et al.; CUREiHUS study group. Proposal for individualized dosing of eculizumab in atypical haemolytic uraemic syndrome: patient friendly and cost-effective. Nephrol Dial Transplant. 2023; 38(2): 362-71. http://doi.org/10.1093/ndt/gfac056.
13. Loschi M, Porcher R, Barraco F et al. Impact of eculizumab treatment on paroxysmal nocturnal hemoglobinuria: a treatment versus no-treatment study. Am J Hematol. 2016; 91(4): 366-70. http://doi.org/10.1002/ajh.24278.