Successful use of reduced-intensity conditioning and matched-unrelated hematopoietic stem cell transplant in a child with Diamond-Blackfan anemia and cirrhosis
Justin M. Asquith
Department of Pediatrics, Nationwide Children's Hospital, Columbus, OH, USA
Department of Internal Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA
Search for more papers by this authorJessica Copacia
Department of Hematology/Oncology/BMT, Nationwide Children's Hospital, Columbus, OH, USA
Search for more papers by this authorMark J. Mogul
Department of Pediatric Hematology and Oncology, Marshall University School of Medicine, Huntington, WV, USA
Search for more papers by this authorCorresponding Author
Rajinder P. S. Bajwa
Department of Hematology/Oncology/BMT, Nationwide Children's Hospital, Columbus, OH, USA
Rajinder P. S. Bajwa, MD, Department of Hematology/Oncology/BMT, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA
Tel.: 614 722 3505
Fax: 614 722 3699
E-mail: [email protected]
Search for more papers by this authorJustin M. Asquith
Department of Pediatrics, Nationwide Children's Hospital, Columbus, OH, USA
Department of Internal Medicine, The Ohio State University Wexner Medical Center, Columbus, OH, USA
Search for more papers by this authorJessica Copacia
Department of Hematology/Oncology/BMT, Nationwide Children's Hospital, Columbus, OH, USA
Search for more papers by this authorMark J. Mogul
Department of Pediatric Hematology and Oncology, Marshall University School of Medicine, Huntington, WV, USA
Search for more papers by this authorCorresponding Author
Rajinder P. S. Bajwa
Department of Hematology/Oncology/BMT, Nationwide Children's Hospital, Columbus, OH, USA
Rajinder P. S. Bajwa, MD, Department of Hematology/Oncology/BMT, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA
Tel.: 614 722 3505
Fax: 614 722 3699
E-mail: [email protected]
Search for more papers by this authorAbstract
For patients with DBA who are transfusion dependent, HSCT is the only cure. Chronic transfusions can lead to cirrhosis secondary to iron overload, making them poor candidates for myeloablative HSCT. RIC regimens are associated with lower morbidity and mortality compared to myeloablative regimens, but use of RIC in DBA has been limited. Here we present a 14-yr-old girl with DBA and multiple comorbidities including liver cirrhosis, who underwent MUD HSCT utilizing a RIC regimen that is novel to this condition. She tolerated the regimen well, and at 21 months, she remains transfusion independent with chimerisms at 99%.
References
- 1Dutt S, Narla A, Lin K, et al. Haploinsufficiency for ribosomal protein genes causes selective activation of p53 in human erythroid progenitor cells. Blood 2011: 117: 2567–2576.
- 2Narla A, Vlachos A, Nathan DG. Diamond Blackfan anemia treatment: Past, present, and future. Semin Hematol 2011: 48: 117–123. Review.
- 3Vlachos A, Muir E. How I treat Diamond-Blackfan anemia. Blood 2010: 116: 3715–3723. Review.
- 4Arnaout K, Patel N, Jain M, El-Amm J, Amro F, Tabbara IA. Complications of allogeneic hematopoietic stem cell transplantation. Cancer Invest 2014: 32: 349–362.
- 5Shenoy S, Grossman WJ, DiPersio J, et al. A novel reduced-intensity stem cell transplant regimen for nonmalignant disorders. Bone Marrow Transplant 2005: 35: 345–352.
- 6Diaconescu R, Flowers CR, Storer B, et al. Morbidity and mortality with nonmyeloablative compared with myeloablative conditioning before hematopoietic cell transplantation from HLA-matched related donors. Blood 2004: 104: 1550–1558.
- 7Morado M, Ojeda E, Garcia-Bustos J, et al. BMT: Serum ferritin as risk factor for veno-occlusive disease of the liver. Prospective Cohort Study. Hematology 2000: 4: 505–512.
- 8Roy V, Perez WS, Eapen M, et al. Bone marrow transplantation for diamond-blackfan anemia. Biol Blood Marrow Transplant 2005: 11: 600–608.
- 9Lipton JM, Ellis SR. Diamond-Blackfan anemia: Diagnosis, treatment, and molecular pathogenesis. Hematol Oncol Clin North Am 2009: 23: 261–282. Review.
- 10Fagioli F, Quarello P, Zecca M, et al. Haematopoietic stem cell transplantation for Diamond Blackfan anaemia: A report from the Italian Association of Paediatric Haematology and Oncology Registry. Br J Haematol 2014: 165: 673–681.
- 11Mugishima H, Ohga S, Ohara A, Kojima S, Fujisawa K, Tsukimoto I; Aplastic Anemia Committee of the Japanese Society of Pediatric Hematology. Hematopoietic stem cell transplantation for Diamond-Blackfan anemia: A report from the Aplastic Anemia Committee of the Japanese Society of Pediatric Hematology. Pediatr Transplant 2007: 11: 601–607.
- 12Pullarkat V, Blanchard S, Tegtmeier B, et al. Iron overload adversely affects outcome of allogeneic hematopoietic cell transplantation. Bone Marrow Transplant 2008: 42: 799–805.
- 13Sorror ML, Maris MB, Storer B, et al. Comparing morbidity and mortality of HLA-matched unrelated donor hematopoietic cell transplantation after nonmyeloablative and myeloablative conditioning: Influence of pretransplantation comorbidities. Blood 2004: 104: 961–968.
- 14Pulsipher MA, Boucher KM, Wall D, et al. Reduced-intensity allogeneic transplantation in pediatric patients ineligible for myeloablative therapy: Results of the Pediatric Blood and Marrow Transplant Consortium Study ONC0313. Blood 2009: 114: 1429–1436.
- 15Parikh SH, Mendizabal A, Benjamin CL, et al. A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases. Biol Blood Marrow Transplant 2014: 20: 326–336.
- 16Tabata S, Mori M, Nagai Y, et al. Successful allogeneic bone marrow transplantation for Diamond-Blackfan anemia complicated by severe cardiac dysfunction due to transfusion-induced hemochromatosis. Intern Med 2010: 49: 453–456.
- 17Ostronoff M, Florencio R, Campos G, et al. Successful nonmyeloablative bone marrow transplantation in a corticosteroid-resistant infant with Diamond-Blackfan anemia. Bone Marrow Transplant 2004: 34: 371–372.
- 18Gómez-Almaguer D, Ruiz-Argüelles GJ, Tarín-Arzaga Ldel C, et al. Reduced-intensity stem cell transplantation in children and adolescents: The Mexican experience. Biol Blood Marrow Transplant 2003; 9: 157–161.
- 19Berndt A, Helwig A, Ehninger G, Bornhäuser M. Successful transplantation of CD34+ selected peripheral blood stem cells from an unrelated donor in an adult patient with Diamond-Blackfan anemia and secondary hemochromatosis. Bone Marrow Transplant 2005: 35: 99–100.
- 20Bizzetto R, Bonfim C, Rocha V, et al.; Eurocord and SAA-WP from EBMT. Outcomes after related and unrelated umbilical cord blood transplantation for hereditary bone marrow failure syndromes other than Fanconi anemia. Haematologica 2011; 96: 134–141.
- 21Lipton JM, Atsidaftos E, Zyskind I, Vlachos A. Improving clinical care and elucidating the pathophysiology of Diamond Blackfan anemia: An update from the Diamond Blackfan Anemia Registry. Pediatr Blood Cancer 2006: 46: 558–564.
- 22Bhatla D, Davies SM, Shenoy S, et al. Reduced-intensity conditioning is effective and safe for transplantation of patients with Shwachman-Diamond syndrome. Bone Marrow Transplant 2008: 42: 159–165.
- 23Woods G, Bajwa RP, Rose MJ. Reduced intensity transplantation for congenital amegakaryocytic thrombocytopenia: Report of a case and review of the literature. Pediatr Transplant 2014: 18: E31–E34.
Citing Literature
