Second allogeneic stem cell transplantation can rescue a significant proportion of patients with JMML relapsing after first allograft

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00064203%3A_____%2F23%3A10457372" target="_blank" >RIV/00064203:_____/23:10457372 - isvavai.cz</a>

Nalezeny alternativní kódy

RIV/00216208:11130/23:10457372

Výsledek na webu

<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=71XXil1WZc" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=71XXil1WZc</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1038/s41409-023-01942-4" target="_blank" >10.1038/s41409-023-01942-4</a>

Jazyk výsledku

angličtina

Název v původním jazyce

Second allogeneic stem cell transplantation can rescue a significant proportion of patients with JMML relapsing after first allograft

Popis výsledku v původním jazyce

Juvenile myelomonocytic leukemia (JMML) is a rare myeloproliferative disease of early childhood. More than 90% of patients harbor mutations in PTPN11, KRAS, NRAS, CBL, or NF1. For most patients, allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative therapy, while relapse is the major cause of treatment failure recorded in about 35% of patients. Age &gt;=2 years, high hemoglobin F (HbF), secondary clonal aberrations, and DNA hypermethylation are associated with an increased risk of relapse. Treatment options for recurrent JMML are limited; for patients still on immunosuppressive therapy, discontinuation of all immunosuppressing agents is generally the first intervention. Donor leukocyte infusions (DLI) can induce a response in some relapsed patients, but the overall outcome of DLI as single strategy has been unfavorable. Few data are available on the efficacy and safety of second HSCT in JMML. We analyzed the outcome of 68 children with JMML who relapsed after first HSCT and received a second allograft.

Název v anglickém jazyce

Second allogeneic stem cell transplantation can rescue a significant proportion of patients with JMML relapsing after first allograft

Popis výsledku anglicky

Juvenile myelomonocytic leukemia (JMML) is a rare myeloproliferative disease of early childhood. More than 90% of patients harbor mutations in PTPN11, KRAS, NRAS, CBL, or NF1. For most patients, allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative therapy, while relapse is the major cause of treatment failure recorded in about 35% of patients. Age &gt;=2 years, high hemoglobin F (HbF), secondary clonal aberrations, and DNA hypermethylation are associated with an increased risk of relapse. Treatment options for recurrent JMML are limited; for patients still on immunosuppressive therapy, discontinuation of all immunosuppressing agents is generally the first intervention. Donor leukocyte infusions (DLI) can induce a response in some relapsed patients, but the overall outcome of DLI as single strategy has been unfavorable. Few data are available on the efficacy and safety of second HSCT in JMML. We analyzed the outcome of 68 children with JMML who relapsed after first HSCT and received a second allograft.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30205 - Hematology

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2023

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

Bone Marrow Transplantation

ISSN

0268-3369

e-ISSN

1476-5365

Svazek periodika

58

Číslo periodika v rámci svazku

5

Stát vydavatele periodika

GB - Spojené království Velké Británie a Severního Irska

Počet stran výsledku

3

Strana od-do

607-609

Kód UT WoS článku

000938041700001

EID výsledku v databázi Scopus

2-s2.0-85148600059