Blinatumomab as a bridge therapy for hematopoietic stem cell transplantation in pediatric refractory/relapsed acute lymphoblastic leukemia

2022
journal article
article
15
cris.lastimport.wos2024-04-09T19:28:37Z
dc.abstract.enDespite the progress that has been made in recent decades in the treatment of pediatric acute leukemias, e.g., converting acute lymphoblastic leukemia (ALL) from a fatal to a highly curable disease, 15–20% of children still relapse. Blinatumomab, a bispecific CD3/CD19 antibody construct, has been successfully used in relapsed/refractory r/r B-cell precursor ALL (BCP-ALL) as a bridge to hematopoietic stem cell transplantation (HSCT). We retrospectively assessed the efficacy and toxicity of blinatumomab in 13 children with r/r BCP-ALL. Between 2017 and 2021, thirteen children, aged 1–18 years, with r/r BCP-ALL were treated with blinatumomab. Two patients were administered blinatumomab for refractory relapse without complete remission (CR), one due to primary refractory disease, and ten patients were in CR with minimal residual disease (MRD) ≥ 10−3. The response rate in our cohort of patients was 85%, with subsequent feasible HSCT in 11 out of 13 children. Ten children reached MRD negativity after the first blinatumomab administration. The three-year OS for the study patients was 85% (Mantel–Cox, p < 0.001) and median follow-up was 24.5 (range: 1–47). All responders proceeded to HSCT and are alive in CR, and MRD negative. Although our study had some limitations with regard to its retrospective design and limited patient population, it clearly showed blinatumomab as not only a feasible but also an effective therapeutic option in pretreated children with r/r BCP-ALL, with a tolerable toxicity profile, paving the way for an HSCT procedure.
dc.affiliationWydział Lekarski : Instytut Pediatriipl
dc.cm.id107116
dc.cm.idOmegaUJCM61f412633dce4488a6e8bfa4ab74b63cpl
dc.contributor.authorPawińska-Wąsikowska, Katarzyna - 214175 pl
dc.contributor.authorWieczorek, Aleksandra - 133763 pl
dc.contributor.authorBalwierz, Walentyna - 128644 pl
dc.contributor.authorBukowska-Straková, Karolina - 173908 pl
dc.contributor.authorSurman, Marta - 137396 pl
dc.contributor.authorSkoczeń, Szymon - 133404 pl
dc.date.accession2022-02-15pl
dc.date.accessioned2022-03-08T08:29:09Z
dc.date.available2022-03-08T08:29:09Z
dc.date.issued2022pl
dc.date.openaccess0
dc.description.accesstimew momencie opublikowania
dc.description.number2pl
dc.description.versionostateczna wersja wydawcy
dc.description.volume14pl
dc.identifier.articleid458pl
dc.identifier.doi10.3390/cancers14020458pl
dc.identifier.eissn2072-6694pl
dc.identifier.issn2072-6694pl
dc.identifier.urihttps://ruj.uj.edu.pl/xmlui/handle/item/288862
dc.identifier.weblinkhttps://www.mdpi.com/2072-6694/14/2/458pl
dc.languageengpl
dc.language.containerengpl
dc.pbn.affiliationDziedzina nauk medycznych i nauk o zdrowiu : nauki medyczne
dc.rightsUdzielam licencji. Uznanie autorstwa 4.0 Międzynarodowa
dc.rights.licenceCC-BY
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/legalcode.pl
dc.share.typeOtwarte czasopismo
dc.subject.plblinatumomabpl
dc.subject.placute lymphoblastic leukemiapl
dc.subject.plimmunotherapypl
dc.subject.plminimal residual diseasepl
dc.subject.plchildrenpl
dc.subtypeArticlepl
dc.titleBlinatumomab as a bridge therapy for hematopoietic stem cell transplantation in pediatric refractory/relapsed acute lymphoblastic leukemiapl
dc.title.journalCancerspl
dc.typeJournalArticlepl
dspace.entity.typePublication
cris.lastimport.wos
2024-04-09T19:28:37Z
dc.abstract.en
Despite the progress that has been made in recent decades in the treatment of pediatric acute leukemias, e.g., converting acute lymphoblastic leukemia (ALL) from a fatal to a highly curable disease, 15–20% of children still relapse. Blinatumomab, a bispecific CD3/CD19 antibody construct, has been successfully used in relapsed/refractory r/r B-cell precursor ALL (BCP-ALL) as a bridge to hematopoietic stem cell transplantation (HSCT). We retrospectively assessed the efficacy and toxicity of blinatumomab in 13 children with r/r BCP-ALL. Between 2017 and 2021, thirteen children, aged 1–18 years, with r/r BCP-ALL were treated with blinatumomab. Two patients were administered blinatumomab for refractory relapse without complete remission (CR), one due to primary refractory disease, and ten patients were in CR with minimal residual disease (MRD) ≥ 10−3. The response rate in our cohort of patients was 85%, with subsequent feasible HSCT in 11 out of 13 children. Ten children reached MRD negativity after the first blinatumomab administration. The three-year OS for the study patients was 85% (Mantel–Cox, p < 0.001) and median follow-up was 24.5 (range: 1–47). All responders proceeded to HSCT and are alive in CR, and MRD negative. Although our study had some limitations with regard to its retrospective design and limited patient population, it clearly showed blinatumomab as not only a feasible but also an effective therapeutic option in pretreated children with r/r BCP-ALL, with a tolerable toxicity profile, paving the way for an HSCT procedure.
dc.affiliationpl
Wydział Lekarski : Instytut Pediatrii
dc.cm.id
107116
dc.cm.idOmegapl
UJCM61f412633dce4488a6e8bfa4ab74b63c
dc.contributor.authorpl
Pawińska-Wąsikowska, Katarzyna - 214175
dc.contributor.authorpl
Wieczorek, Aleksandra - 133763
dc.contributor.authorpl
Balwierz, Walentyna - 128644
dc.contributor.authorpl
Bukowska-Straková, Karolina - 173908
dc.contributor.authorpl
Surman, Marta - 137396
dc.contributor.authorpl
Skoczeń, Szymon - 133404
dc.date.accessionpl
2022-02-15
dc.date.accessioned
2022-03-08T08:29:09Z
dc.date.available
2022-03-08T08:29:09Z
dc.date.issuedpl
2022
dc.date.openaccess
0
dc.description.accesstime
w momencie opublikowania
dc.description.numberpl
2
dc.description.version
ostateczna wersja wydawcy
dc.description.volumepl
14
dc.identifier.articleidpl
458
dc.identifier.doipl
10.3390/cancers14020458
dc.identifier.eissnpl
2072-6694
dc.identifier.issnpl
2072-6694
dc.identifier.uri
https://ruj.uj.edu.pl/xmlui/handle/item/288862
dc.identifier.weblinkpl
https://www.mdpi.com/2072-6694/14/2/458
dc.languagepl
eng
dc.language.containerpl
eng
dc.pbn.affiliation
Dziedzina nauk medycznych i nauk o zdrowiu : nauki medyczne
dc.rights
Udzielam licencji. Uznanie autorstwa 4.0 Międzynarodowa
dc.rights.licence
CC-BY
dc.rights.uri
http://creativecommons.org/licenses/by/4.0/legalcode.pl
dc.share.type
Otwarte czasopismo
dc.subject.plpl
blinatumomab
dc.subject.plpl
acute lymphoblastic leukemia
dc.subject.plpl
immunotherapy
dc.subject.plpl
minimal residual disease
dc.subject.plpl
children
dc.subtypepl
Article
dc.titlepl
Blinatumomab as a bridge therapy for hematopoietic stem cell transplantation in pediatric refractory/relapsed acute lymphoblastic leukemia
dc.title.journalpl
Cancers
dc.typepl
JournalArticle
dspace.entity.type
Publication
Affiliations

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