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Review

Difficult Cases of Paroxysmal Nocturnal Hemoglobinuria: Diagnosis and Therapeutic Novelties

1
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, University of Milan, 20122 Milan, Italy
2
Department of Oncology and Oncohematology, University of Milan, 20122 Milan, Italy
*
Author to whom correspondence should be addressed.
Academic Editor: Fernando Ramos
J. Clin. Med. 2021, 10(5), 948; https://doi.org/10.3390/jcm10050948
Received: 28 January 2021 / Revised: 14 February 2021 / Accepted: 20 February 2021 / Published: 1 March 2021
(This article belongs to the Special Issue Diagnostics and Management of Myelodysplastic Syndromes)
Paroxysmal nocturnal hemoglobinuria (PNH) is an intriguing disease that can pose many difficulties to physicians, as well as to hematologists, who are unfamiliar with it. Research regarding its pathophysiologic, diagnostic, and therapeutic aspects is still ongoing. In the last ten years, new flow cytometry techniques with high sensitivity enabled us to detect PNH clones as small as <1% of a patient’s hematopoiesis, resulting in increasing incidence but more difficult data interpretation. Particularly, the clinical significance of small PNH clones in patients with bone marrow failures, including aplastic anemia and myelodysplastic syndromes, as well as in uncommon associations, such as myeloproliferative disorders, is still largely unknown. Besides current treatment with the anti-C5 eculizumab, which reduced PNH-related morbidity and mortality, new complement inhibitors will likely fulfill unmet clinical needs in terms of patients’ quality of life and better response rates (i.e., responses in subjects with C5 polymorphisms; reduction of extravascular hemolysis and breakthrough hemolysis episodes). Still, unanswered questions remain for these agents regarding their use in mono- or combination therapy, when to treat, and which drug is the best for which patient. Lastly, long-term safety needs to be assessed in real-life studies. In this review, we describe some clinical vignettes illustrating practical aspects of PNH diagnosis and management; moreover, we discuss recent advances in PNH diagnostic and therapeutic approaches. View Full-Text
Keywords: paroxysmal nocturnal hemoglobinuria; eculizumab; complement inhibitors; bone marrow failures; myelodysplastic syndromes paroxysmal nocturnal hemoglobinuria; eculizumab; complement inhibitors; bone marrow failures; myelodysplastic syndromes
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MDPI and ACS Style

Fattizzo, B.; Serpenti, F.; Giannotta, J.A.; Barcellini, W. Difficult Cases of Paroxysmal Nocturnal Hemoglobinuria: Diagnosis and Therapeutic Novelties. J. Clin. Med. 2021, 10, 948. https://doi.org/10.3390/jcm10050948

AMA Style

Fattizzo B, Serpenti F, Giannotta JA, Barcellini W. Difficult Cases of Paroxysmal Nocturnal Hemoglobinuria: Diagnosis and Therapeutic Novelties. Journal of Clinical Medicine. 2021; 10(5):948. https://doi.org/10.3390/jcm10050948

Chicago/Turabian Style

Fattizzo, Bruno, Fabio Serpenti, Juri Alessandro Giannotta, and Wilma Barcellini. 2021. "Difficult Cases of Paroxysmal Nocturnal Hemoglobinuria: Diagnosis and Therapeutic Novelties" Journal of Clinical Medicine 10, no. 5: 948. https://doi.org/10.3390/jcm10050948

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