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Successful allogeneic bone marrow transplantation using immunosuppressive conditioning regimen for a patient with red blood cell transfusiondependent pyruvate kinase deficiency anemia
Pyruvate kinase deficiency (PKD) is the rare glycolytic enzyme defect causing hemolytic anemia. Treatments are mainly red cell transfusion and/or splenectomy, leading to iron overload. Allogeneic bone marrow transplantation (BMT) is alternatively curative treatment for severe PKD. The intensity of c...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
PAGEPress Publications, Pavia, Italy
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7256629/ https://www.ncbi.nlm.nih.gov/pubmed/32499904 http://dx.doi.org/10.4081/hr.2020.8305 |
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author | Shimomura, Maiko Doi, Takehiko Nishimura, Shiho Imanaka, Yusuke Karakawa, Shuhei Okada, Satoshi Kawaguchi, Hiroshi Kobayashi, Masao |
author_facet | Shimomura, Maiko Doi, Takehiko Nishimura, Shiho Imanaka, Yusuke Karakawa, Shuhei Okada, Satoshi Kawaguchi, Hiroshi Kobayashi, Masao |
author_sort | Shimomura, Maiko |
collection | PubMed |
description | Pyruvate kinase deficiency (PKD) is the rare glycolytic enzyme defect causing hemolytic anemia. Treatments are mainly red cell transfusion and/or splenectomy, leading to iron overload. Allogeneic bone marrow transplantation (BMT) is alternatively curative treatment for severe PKD. The intensity of conditioning is often controversial because of higher risk of graft failure and organ damage. Here, we present a transfusion-dependent PKD patient undergoing BMT from an HLA-identical sibling using intensively immunosuppressive conditioning regimen. This report suggests that BMT using immunosuppressive conditioning regimen may be a feasible and effective treatment for patients with severe PKD with iron overload. We suggest the timing of the transplantation at an earlier age in severe PKD predicted from gene mutation is preferred before cumulative damage of transfusion. |
format | Online Article Text |
id | pubmed-7256629 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | PAGEPress Publications, Pavia, Italy |
record_format | MEDLINE/PubMed |
spelling | pubmed-72566292020-06-03 Successful allogeneic bone marrow transplantation using immunosuppressive conditioning regimen for a patient with red blood cell transfusiondependent pyruvate kinase deficiency anemia Shimomura, Maiko Doi, Takehiko Nishimura, Shiho Imanaka, Yusuke Karakawa, Shuhei Okada, Satoshi Kawaguchi, Hiroshi Kobayashi, Masao Hematol Rep Case Report Pyruvate kinase deficiency (PKD) is the rare glycolytic enzyme defect causing hemolytic anemia. Treatments are mainly red cell transfusion and/or splenectomy, leading to iron overload. Allogeneic bone marrow transplantation (BMT) is alternatively curative treatment for severe PKD. The intensity of conditioning is often controversial because of higher risk of graft failure and organ damage. Here, we present a transfusion-dependent PKD patient undergoing BMT from an HLA-identical sibling using intensively immunosuppressive conditioning regimen. This report suggests that BMT using immunosuppressive conditioning regimen may be a feasible and effective treatment for patients with severe PKD with iron overload. We suggest the timing of the transplantation at an earlier age in severe PKD predicted from gene mutation is preferred before cumulative damage of transfusion. PAGEPress Publications, Pavia, Italy 2020-05-15 /pmc/articles/PMC7256629/ /pubmed/32499904 http://dx.doi.org/10.4081/hr.2020.8305 Text en ©Copyright: the Author(s) http://creativecommons.org/licenses/by-nc/4.0/ This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/4.0/) which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Case Report Shimomura, Maiko Doi, Takehiko Nishimura, Shiho Imanaka, Yusuke Karakawa, Shuhei Okada, Satoshi Kawaguchi, Hiroshi Kobayashi, Masao Successful allogeneic bone marrow transplantation using immunosuppressive conditioning regimen for a patient with red blood cell transfusiondependent pyruvate kinase deficiency anemia |
title | Successful allogeneic bone marrow transplantation using immunosuppressive conditioning regimen for a patient with red blood cell transfusiondependent pyruvate kinase deficiency anemia |
title_full | Successful allogeneic bone marrow transplantation using immunosuppressive conditioning regimen for a patient with red blood cell transfusiondependent pyruvate kinase deficiency anemia |
title_fullStr | Successful allogeneic bone marrow transplantation using immunosuppressive conditioning regimen for a patient with red blood cell transfusiondependent pyruvate kinase deficiency anemia |
title_full_unstemmed | Successful allogeneic bone marrow transplantation using immunosuppressive conditioning regimen for a patient with red blood cell transfusiondependent pyruvate kinase deficiency anemia |
title_short | Successful allogeneic bone marrow transplantation using immunosuppressive conditioning regimen for a patient with red blood cell transfusiondependent pyruvate kinase deficiency anemia |
title_sort | successful allogeneic bone marrow transplantation using immunosuppressive conditioning regimen for a patient with red blood cell transfusiondependent pyruvate kinase deficiency anemia |
topic | Case Report |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7256629/ https://www.ncbi.nlm.nih.gov/pubmed/32499904 http://dx.doi.org/10.4081/hr.2020.8305 |
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