Paroxysmal nocturnal hemoglobinuria medical therapy
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief:
Overview
There is no treatment for [disease name]; the mainstay of therapy is supportive care.
OR
Supportive therapy for [disease name] includes [therapy 1], [therapy 2], and [therapy 3].
OR
The majority of cases of [disease name] are self-limited and require only supportive care.
OR
[Disease name] is a medical emergency and requires prompt treatment.
OR
The mainstay of treatment for [disease name] is [therapy].
OR The optimal therapy for [malignancy name] depends on the stage at diagnosis.
OR
[Therapy] is recommended among all patients who develop [disease name].
OR
Pharmacologic medical therapy is recommended among patients with [disease subclass 1], [disease subclass 2], and [disease subclass 3].
OR
Pharmacologic medical therapies for [disease name] include (either) [therapy 1], [therapy 2], and/or [therapy 3].
OR
Empiric therapy for [disease name] depends on [disease factor 1] and [disease factor 2].
OR
Patients with [disease subclass 1] are treated with [therapy 1], whereas patients with [disease subclass 2] are treated with [therapy 2].
Medical Therapy
- The mainstay of treatment for PNH is medical therapy.
- Treatment of PNH includes the following:
- Treatment of the anemia
- Anti-complement therapy
- Hematopoietic cell transplantation
Treatment of the anemia
Hematopoietic cell transplantation
- Hematopoietic stem cell transplantation is an important curative therapy for paroxysmal nocturnal hemoglobinuria.
- Stem cell transplantation is usually reserved for the severely affected patients and is indicated for the following type of patients:[1]
- Patients with severe aplastic anemia who are HLA matched donor.
- Patients with myelodysplastic syndromes
- Patients who are unresponsive to the anti complement therapy (eculizumab)
- Transplantation related issues:[2][3]
- Acute Gravt-Versus-Host Disease (GVHD) occurs in almost third of the PNH patients treated with transplant.
- Survival rate of the PNH patients treated with transplantation is from 50% to 60%.
References
- ↑ DeZern AE, Zahurak M, Symons H, Cooke K, Jones RJ, Brodsky RA (2017). "Alternative Donor Transplantation with High-Dose Post-Transplantation Cyclophosphamide for Refractory Severe Aplastic Anemia". Biol Blood Marrow Transplant. 23 (3): 498–504. doi:10.1016/j.bbmt.2016.12.628. PMC 5373094. PMID 28013015.
- ↑ Peffault de Latour R, Schrezenmeier H, Bacigalupo A, Blaise D, de Souza CA, Vigouroux S; et al. (2012). "Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria". Haematologica. 97 (11): 1666–73. doi:10.3324/haematol.2012.062828. PMC 3487438. PMID 22689687.
- ↑ Parker C, Omine M, Richards S, Nishimura J, Bessler M, Ware R; et al. (2005). "Diagnosis and management of paroxysmal nocturnal hemoglobinuria". Blood. 106 (12): 3699–709. doi:10.1182/blood-2005-04-1717. PMC 1895106. PMID 16051736.