Outcome of allogeneic hematopoietic stem cell transplantation in adult patients with paroxysmal nocturnal hemoglobinuria.
Adolescent
Adult
Age Factors
Blood Transfusion
Databases as Topic
Female
Hematopoietic Stem Cell Transplantation
/ methods
Hemoglobinuria, Paroxysmal
/ mortality
Humans
Male
Middle Aged
Preoperative Care
Registries
Retrospective Studies
Survival Rate
Transplantation, Homologous
Treatment Outcome
Young Adult
Allogeneic hematopoietic stem cell transplantation
Paroxysmal nocturnal hemoglobinuria
Patient age
Pretransplant transfusion
Prognostic factor
Journal
International journal of hematology
ISSN: 1865-3774
Titre abrégé: Int J Hematol
Pays: Japan
ID NLM: 9111627
Informations de publication
Date de publication:
Jan 2021
Jan 2021
Historique:
received:
30
05
2020
accepted:
25
08
2020
revised:
25
07
2020
pubmed:
6
9
2020
medline:
12
2
2021
entrez:
5
9
2020
Statut:
ppublish
Résumé
The safety and efficacy of allogeneic hematopoietic stem cell transplantation (HSCT) for paroxysmal nocturnal hemoglobinuria (PNH) remain unclear. Therefore, we retrospectively analyzed the outcomes of 42 adult patients with PNH who underwent allogeneic HSCT using the registry database of the Japan Society for Hematopoietic Cell Transplantation. The median patient age was 32.5 years. The number of packed red cell (PRC) transfusions was < 20 times in 19 patients and ≥ 20 times in 16; 7 patients had missing data. Stem cell sources were bone marrow (N = 15) or peripheral blood (N = 13) from a related donor or bone marrow (N = 11) and cord blood (N = 3) from an unrelated donor. The cumulative incidence of neutrophil engraftment at day 40 was 81%. Six patients died before engraftment, and the 6-year overall survival (OS) was 74%. The OS of patients with < 20 pretransplant PRC transfusions was significantly higher than that of patients with ≥ 20 pretransplant PRC transfusions (95% vs. 63%; P < 0.05). Moreover, the OS of patients aged < 30 years was significantly higher than that of patients aged ≥ 30 years (90% vs. 59%; P < 0.05). Allogeneic HSCT for PNH could provide favorable survival; however, pretransplant transfusion burden and patient age should be considered when deciding the timing of allogeneic HSCT.
Identifiants
pubmed: 32889696
doi: 10.1007/s12185-020-02982-y
pii: 10.1007/s12185-020-02982-y
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
122-127Subventions
Organisme : Japan Agency for Medical Research and Development (JP)
ID : 19ek0510023h0002
Références
Hill A, DeZern AE, Kinoshita T, Brodsky RA. Paroxysmal nocturnal haemoglobinuria. Nat Rev Dis Primers. 2017;3:17028.
doi: 10.1038/nrdp.2017.28
Devos T, Meers S, Boeckx N, Gothot A, Deeren D, Chatelain B, et al. Diagnosis and management of PNH: review and recommendations from a Belgian expert panel. Eur J Haematol. 2018;101:737–49.
doi: 10.1111/ejh.13166
Kelly RJ, Hill A, Arnold LM, Brooksbank GL, Richards SJ, Cullen M, et al. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. 2011;117:6786–92.
doi: 10.1182/blood-2011-02-333997
Ueda Y, Obara N, Yonemura Y, Noji H, Masuko M, Seki Y, et al. Effects of eculizumab treatment on quality of life in patients with paroxysmal nocturnal hemoglobinuria in Japan. Int J Hematol. 2018;107:656–65.
doi: 10.1007/s12185-018-2409-3
Saso R, Marsh J, Cevreska L, Szer J, Gale RP, Rowlings PA, et al. Bone marrow transplants for paroxysmal nocturnal haemoglobinuria. Br J Haematol. 1999;104:392–6.
doi: 10.1046/j.1365-2141.1999.01195.x
Srinivasan R, Takahashi Y, McCoy JP, Espinoza-Delgado I, Dorrance C, Igarashi T, et al. Overcoming graft rejection in heavily transfused and allo-immunised patients with bone marrow failure syndromes using fludarabine-based haematopoietic cell transplantation. Br J Haematol. 2006;133:305–14.
doi: 10.1111/j.1365-2141.2006.06019.x
Santarone S, Bacigalupo A, Risitano AM, Tagliaferri E, Di Bartolomeo E, Iori AP, et al. Hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria: long-term results of a retrospective study on behalf of the Gruppo Italiano Trapianto Midollo Osseo (GITMO). Haematologica. 2010;95:983–8.
doi: 10.3324/haematol.2009.017269
de Latour RP, Schrezenmeier H, Bacigalupo A, Blaise D, de Souza CA, Vigouroux S, et al. Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria. Haematologica. 2012;97:1666–733.
doi: 10.3324/haematol.2012.062828
Pantin J, Tian X, Geller N, Ramos C, Cook L, Cho E, et al. Long-term outcome of fludarabine-based reduced-intensity allogeneic hematopoietic cell transplantation for debilitating paroxysmal nocturnal hemoglobinuria. Biol Blood Marrow Transplant. 2014;20:1435–9.
doi: 10.1016/j.bbmt.2014.05.012
Kamranzadeh Fumani H, Zokaasadi M, Kasaeian A, Alimoghaddam K, Mousavi SA, Bahar B, et al. Allogeneic hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria: a retrospective single-center study. Hematol Oncol. 2017;35:935–8.
doi: 10.1002/hon.2367
Lee SE, Park SS, Jeon YW, Yoon JH, Cho BS, Eom KS, et al. Outcomes of allogeneic stem cell transplantation in patients with paroxysmal nocturnal hemoglobinuria with or without aplastic anemia. Eur J Haematol. 2017;99:336–43.
doi: 10.1111/ejh.12922
Vallet N, de Fontbrune FS, Loschi M, Desmier D, Villate A, Barraco F, et al. Hematopoietic stem cell transplantation for patients with paroxysmal nocturnal hemoglobinuria previously treated with eculizumab: a retrospective study of 21 patients from SFGM-TC centers. Haematologica. 2018;103:e103–e105105.
doi: 10.3324/haematol.2017.182360
Cooper JP, Farah RJ, Stevenson PA, Gooley TA, Storb R, Scott BL. Hematopoietic cell transplantation for paroxysmal nocturnal hemoglobinuria in the age of eculizumab. Biol Blood Marrow Transplant. 2019;25:1331–9.
doi: 10.1016/j.bbmt.2019.01.033
Liu L, Liu S, Zhang Y, Zhou H, Wang Q, Tian H, et al. Excellent outcomes of allogeneic hematopoietic stem cell transplantation in patients with paroxysmal nocturnal hemoglobinuria: a single-center study. Biol Blood Marrow Transplant. 2019;25:1544–9.
doi: 10.1016/j.bbmt.2019.02.024
Markiewicz M, Drozd-Sokolowska J, Biecek P, Dzierzak-Mietla M, Boguradzki P, Staniak M, et al. Allogeneic hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria: multicenter analysis by the Polish Adult Leukemia Group. Biol Blood Marrow Transplant. 2020;20:30347–55.
Atsuta Y. Introduction of Transplant Registry Unified Management Program 2 (TRUMP2): scripts for TRUMP data analyses, part I (variables other than HLA-related data). Int J Hematol. 2016;103:3–10.
doi: 10.1007/s12185-015-1894-x
Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J, et al. 1994 Consensus conference on acute GVHD grading. Bone Marrow Transplant. 1995;15:825–8.
pubmed: 7581076
Shulman HM, Sullivan KM, Weiden PL, McDonald GB, Striker GE, Sale GE, et al. Chronic graft-versus-host syndrome in man. A long-term clinicopathologic study of 20 Seattle patients. Am J Med. 1980;69:204–17.
doi: 10.1016/0002-9343(80)90380-0
Bacigalupo A, Ballen K, Rizzo D, Giralt S, Lazarus H, Ho V, et al. Defining the intensity of conditioning regimens: working definitions. Biol Blood Marrow Transplant. 2009;15:1628–33.
doi: 10.1016/j.bbmt.2009.07.004
Gooley TA, Leisenring W, Crowley J, Storer BE. Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Stat Med. 1999;18:695–706.
doi: 10.1002/(SICI)1097-0258(19990330)18:6<695::AID-SIM60>3.0.CO;2-O
Kanda Y. Investigation of the freely available easy-to-use software 'EZR' for medical statistics. Bone Marrow Transplant. 2013;48:452–8.
doi: 10.1038/bmt.2012.244
Passweg JR, Socié G, Hinterberger W, Bacigalupo A, Biggs JC, Camitta BM, et al. Bone marrow transplantation for severe aplastic anemia: has outcome improved? Blood. 1997;90:858–64.
pubmed: 9226187
Lee SE, Yahng SA, Cho BS, Eom KS, Kim YJ, Kim HJ, et al. Impact of pretransplant red cell transfusion on outcome after allogeneic stem cell transplantation in adult patients with severe aplastic anemia. Bone Marrow Transplant. 2016;51:1323–9.
doi: 10.1038/bmt.2016.140
Armand P, Kim HT, Cutler CS, Ho VT, Koreth J, Alyea EP, et al. Prognostic impact of elevated pretransplantation serum ferritin in patients undergoing myeloablative stem cell transplantation. Blood. 2007;109:4586–8.
doi: 10.1182/blood-2006-10-054924
Kataoka K, Nannya Y, Hangaishi A, Imai Y, Chiba S, Takahashi T, et al. Influence of pretransplantation serum ferritin on nonrelapse mortality after myeloablative and nonmyeloablative allogeneic hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2009;15:195–204.
doi: 10.1016/j.bbmt.2008.11.012
Allali S, de Montalembert M, Brousse V, Chalumeau M, Karim Z. Management of iron overload in hemoglobinopathies. Transfus Clin Biol. 2017;24:223–6.
doi: 10.1016/j.tracli.2017.06.008
DeZern AE, Jones RJ, Brodsky RA. Eculizumab bridging before bone marrow transplant for marrow failure disorders is safe and does not limit engraftment. Biol Blood Marrow Transplant. 2018;24:e26–30.
doi: 10.1016/j.bbmt.2018.07.032
Mei M, Gupta R, O'Donnell M, Al Malki MM, Aldoss I, Ali H, et al. Post-allogeneic hematopoietic stem cell transplantation eculizumab as prophylaxis against hemolysis and thrombosis for patients with hematologic disorders associated with paroxysmal nocturnal hemoglobinuria clones. Biol Blood Marrow Transplant. 2019;25:e183–e185185.
doi: 10.1016/j.bbmt.2019.01.025