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Annals of Hematology
Published in: Annals of Hematology 10/2019

01-10-2019 | Myelodysplastic Syndrome | Letter to the Editor

Successful cord blood transplantation for a paroxysmal nocturnal hemoglobinuria complicated with Budd-Chiari syndrome and myelodysplastic syndrome

Authors: Taro Takahashi, Satoshi Ichikawa, Hideo Harigae

Published in: Annals of Hematology | Issue 10/2019

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Excerpt

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder of hematopoietic stem cells. The disease is characterized by hemolytic anemia, bone marrow failure, and thrombosis [1]. Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) is the only curative treatment for PNH, but it has a disadvantage of high incidence of treatment-related death. Therefore, the indication is currently limited [2]. …
Literature
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Matos-Fernandez NA, Yasser R et al (2009) Current status of allogeneic hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria. Biol Blood Marrow Transplant 15:656–661CrossRefPubMed
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Hillmen P, Muus P, Duhrsen U, Risitano AM, Schubert J, Luzzatto L, Schrezenmeier H, Szer J, Brodsky RA, Hill A, Socie G, Bessler M, Rollins SA, Bell L, Rother RP, Young NS (2007) Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood 110:4123–4128CrossRefPubMed
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Kelly RJ, Hill A, Arnold LM, Brooksbank GL, Richards SJ, Cullen M, Mitchell LD, Cohen DR, Gregory WM, Hillmen P (2011) Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood 117:6786–6792CrossRefPubMed
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Cooper JP, Farah RJ, Stevenson PA, Gooley TA, Storb R, Scott BL (2019) Hematopoietic cell transplantation for paroxysmal nocturnal hemoglobinuria in the age of eculizumab. Biol Blood Marrow Transplant 25:1331–1339CrossRefPubMed
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Saso R, Marsh J, Cevreska L, Szer J, Gale RP, Rowlings P, Passweg J, Nugent M, Luzzatto L, Horowitz M, Gordon-Smith E (1999) Bone marrow transplants for paroxysmal nocturnal haemoglobinuria. Br J Haematol 104:392–396CrossRefPubMed
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de Latour RP, Schrezenmeier H, Bacigalupo A, Blaise D, de Souza CA, Vigouroux S, Willemze R, Terriou L, Tichelli A, Mohty M, de Guibert S, Marsh JC, Passweg J, Yves Mary J, Socie G (2012) Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria. Haematologica 97:1666–1673CrossRefPubMedCentral
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Pantin J, Tian X, Geller N, Ramos C, Cook L, Cho E, Scheinberg P, Vasu S, Khuu H, Stroncek D, Barrett J, Young NS, Donohue T, Childs RW (2014) Long-term outcome of fludarabine-based reduced-intensity allogeneic hematopoietic cell transplantation for debilitating paroxysmal nocturnal hemoglobinuria. Biol Blood Marrow Transplant 20:1435–1454CrossRefPubMedPubMedCentral
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Yamamoto H, Uchida N, Yuasa M, Kageyama K, Ota H, Kaji D, Nishida A, Ishiwata K, Takagi S, Tsuji M, Asano-Mori Y, Yamamoto G, Izutsu K, Masuoka K, Wake A, Yoneyama A, Makino S, Taniguchi S (2016) A novel reduced-toxicity myeloablative conditioning regimen using full-dose busulfan, fludarabine, and melphalan for single cord blood transplantation provides durable engraftment and remission in nonremission myeloid malignancies. Biol Blood Marrow Transplant 22:1844–1850CrossRefPubMed
Metadata
Title
Successful cord blood transplantation for a paroxysmal nocturnal hemoglobinuria complicated with Budd-Chiari syndrome and myelodysplastic syndrome
Authors
Taro Takahashi
Satoshi Ichikawa
Hideo Harigae
Publication date
01-10-2019
Publisher
Springer Berlin Heidelberg
Published in
Annals of Hematology / Issue 10/2019
Print ISSN: 0939-5555
Electronic ISSN: 1432-0584
DOI
https://doi.org/10.1007/s00277-019-03781-0

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