Outcome of allogeneic hematopoietic stem cell transplantation in adult patients with paroxysmal nocturnal hemoglobinuria View Full Text


Ontology type: schema:ScholarlyArticle     


Article Info

DATE

2020-09-05

AUTHORS

Yukinori Nakamura, Katsuto Takenaka, Hirohito Yamazaki, Yasushi Onishi, Yukiyasu Ozawa, Kazuhiro Ikegame, Ken-ichi Matsuoka, Tomomi Toubai, Yasunori Ueda, Yoshinobu Kanda, Tatsuo Ichinohe, Yoshiko Atsuta, Takehiko Mori

ABSTRACT

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. More... »

PAGES

122-127

Journal

TITLE

International Journal of Hematology

ISSUE

1

VOLUME

113

Identifiers

URI

http://scigraph.springernature.com/pub.10.1007/s12185-020-02982-y

DOI

http://dx.doi.org/10.1007/s12185-020-02982-y

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1130621241

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/32889696


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29 schema:description 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.
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36 OS of patients
37 PRC transfusion
38 adult patients
39 age
40 allogeneic hematopoietic stem cell transplantation
41 blood
42 bone marrow
43 burden
44 cell source
45 cell transfusion
46 cell transplantation
47 cord blood
48 cumulative incidence
49 data
50 database
51 day 40
52 donors
53 efficacy
54 engraftment
55 favorable survival
56 hematopoietic cell transplantation
57 hematopoietic stem cell transplantation
58 hemoglobinuria
59 incidence
60 marrow
61 median patient age
62 neutrophil engraftment
63 nocturnal hemoglobinuria
64 number
65 outcomes
66 overall survival
67 packed red cell transfusion
68 paroxysmal nocturnal hemoglobinuria
69 patient age
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71 peripheral blood
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75 safety
76 society
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78 stem cell transplantation
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80 time
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