Publications by authors named "Perez-Simon J"

Ruxolitinib has been approved for the treatment of adults and pediatric patients ≥12 years with steroid refractory graft-versus-host disease (GvHD). However, real-life studies are needed to confirm the results of clinical trials and further assess its efficacy in special populations. We performed a descriptive, retrospective, multi-center study of 352 adults and 42 pediatric patients treated with ruxolitinib for steroid-refractory acute or chronic GvHD.

View Article and Find Full Text PDF

Background: LAMVYX was a multicenter, single-arm, phase 2 trial designed to validate the safety and efficacy of CPX-351 in patients aged 60-75 years with newly diagnosed, secondary acute myeloid leukemia and to generate evidence on key issues not addressed in the preceding regulatory pivotal trial.

Methods: The primary end point of the study was the complete remission (CR)/CR with incomplete hematologic recovery (CRi) rate after induction. Eligible patients were recommended to undergo allogeneic hematopoietic stem cell transplantation after the first consolidation cycle.

View Article and Find Full Text PDF
Article Synopsis
  • The study investigates the effectiveness of axatilimab, a CSF1R-blocking antibody, for treating chronic graft-versus-host disease (GVHD) in patients post-hematopoietic stem-cell transplantation.
  • In a phase 2 trial involving 241 participants, different doses of axatilimab were tested, with overall response rates of 74%, 67%, and 50% across three dose groups.
  • Alongside substantial improvement in GVHD symptoms measured by patient-reported outcomes, the most frequent side effects were temporary lab abnormalities linked to the treatment.
View Article and Find Full Text PDF
Article Synopsis
  • Immune effector cell-associated hematotoxicity (ICAHT) is a common complication in CAR-T cell therapy, leading to significant thrombocytopenia in about one-third of patients, often necessitating platelet transfusions.
  • Eltrombopag, a thrombopoietin receptor agonist, was evaluated in a study involving 38 patients from 10 hospitals in Spain who developed platelet transfusion dependence after CAR-T treatment.
  • The results showed that 76.3% of patients achieved platelet transfusion independence after using eltrombopag, with additional improvements noted in neutrophil and red blood cell counts, and no serious side effects reported.
View Article and Find Full Text PDF

Although broadly used, consolidative autologous hematopoietic stem cell transplantation (auto-HCT) for relapsed/refractory (R/R) T-cell/histiocyte-rich large B-cell lymphoma (THRLBCL) has never been specifically investigated. Here, we have analyzed outcomes of auto-HCT for THRLBCL compared with diffuse large cell B-cell lymphoma not otherwise specified (DLBCL). Eligible for this retrospective registry study were adult patients with R/R THRLBCL and DLBCL, respectively, who underwent a first auto-HCT in a salvage-sensitive disease status as assessed by positron emission tomography-computed tomography between 2016 and 2021 and were registered with the European Society for Blood and Marrow Transplantation database.

View Article and Find Full Text PDF
Article Synopsis
  • - The addition of molecular data to prognostic models has improved the risk assessment of low-risk myelodysplastic neoplasms (LR-MDS), but the specific impact of molecular lesions in this group remains uncertain.
  • - In a study of 227 LR-MDS patients, RUNX1 mutations were linked to lower overall survival, while SF3B1 mutations showed a reduced risk of death; both TP53 and RUNX1 mutations were also predictive of leukaemic progression.
  • - The traditional measure of blast percentage did not significantly affect survival or progression probabilities in LR-MDS patients when molecular data was included, highlighting its decreased clinical relevance in this context.
View Article and Find Full Text PDF
Article Synopsis
  • CAR therapy targeting BCMA is being improved with the new anti-BCMA CAR called CARTemis-1, which aims to be more effective against multiple myeloma, addressing the challenges of current treatments that lack sustained effectiveness.
  • The study demonstrated that the longer version of CARTemis-1 showed better cancer-killing ability and confirmed that certain manufacturing conditions, like specific cytokines, enhance CAR-T cell quality and effectiveness.
  • CARTemis-1 was found to be effective without being inhibited by soluble BCMA, and it successfully met regulatory standards for production while exhibiting strong antitumor effects in both lab and live models.
View Article and Find Full Text PDF

Objectives: Patients with haematologic malignancies (HM) COVID-19 have more severe disease, with increased risk of mortality. Therefore, this study aimed to evaluate the effect of SARS-CoV-2 RNAemia and the specific humoral immune responses on the clinical outcomes of patients with HM and COVID-19.

Methods: Interferon-α/γ (IFN-α/IFN-γ) serum levels, neutralizing antibodies and RNAemia at COVID-19 diagnosis, and persistent RNAemia during the follow-up were evaluated.

View Article and Find Full Text PDF

Background: There are no studies assessing the evolution and patterns of genetic studies performed at diagnosis in acute myeloid leukemia (AML) patients. Such studies could help to identify potential gaps in our present diagnostic practices, especially in the context of increasingly complex procedures and classifications.

Methods: The REALMOL study (NCT05541224) evaluated the evolution, patterns, and clinical impact of performing main genetic and molecular studies performed at diagnosis in 7285 adult AML patients included in the PETHEMA AML registry (NCT02607059) between 2000 and 2021.

View Article and Find Full Text PDF

FLT3-ITD and NPM1 mutations are key to defining the genetic risk profile of acute myeloid leukemia (AML). We aimed to assess the prognostic features of the FLT3-ITD and NPM1 mutations in old and/or unfit individuals with AML treated with non-intensive therapies in the era before azacitidine-venetoclax approbation. The results of various non-intensive regimens were also compared.

View Article and Find Full Text PDF

Background: Iadademstat is a potent, selective, oral inhibitor of both the enzymatic and scaffolding activities of the transcriptional repressor lysine-specific demethylase 1 (LSD1; also known as KDM1A) that showed promising early activity and safety in a phase 1 trial and strong preclinical synergy with azacitidine in acute myeloid leukaemia cell lines. Therefore, we aimed to investigate the combination of iadademstat and azacitidine for the treatment of adult patients with newly diagnosed acute myeloid leukaemia.

Methods: The open-label, phase 2a, dose-finding ALICE study was conducted at six hospitals in Spain and enrolled patients aged 18 years or older with newly diagnosed acute myeloid leukaemia not eligible for intensive chemotherapy and an ECOG performance status of 0-2.

View Article and Find Full Text PDF
Article Synopsis
  • A study compared the effectiveness of posttransplant cyclophosphamide (PTCy) versus conventional calcineurin inhibitor (CNI)-based regimens for preventing graft-versus-host disease (GVHD) in patients with Hodgkin lymphoma undergoing hematopoietic stem cell transplantation (HSCT) from HLA-matched donors.
  • Results showed that while PTCy was linked to slower recovery of blood cells, it significantly reduced the risk of chronic GVHD and improved long-term survival measures, including overall survival (OS) and progression-free survival (PFS).
  • Overall, PTCy exhibited better outcomes in reducing relapse rates and improving survival compared to CNI-based prophylaxis in this patient group, suggesting it may be a
View Article and Find Full Text PDF

Acute myeloid leukemia (AML) is an aggressive hematologic malignancy with a poor prognosis despite the advent of novel therapies. Consequently, a major need exists for new therapeutic options, particularly for patients with relapsed/refractory (R/R) AML. In recent years, it has been possible to individualize the treatment of a subgroup of patients, particularly with the emergence of multiple targeted therapies.

View Article and Find Full Text PDF

We aimed to compare outcomes following treosulfan (TREO) or busulfan (BU) conditioning in a large cohort of myelofibrosis (MF) patients from the EBMT registry. A total of 530 patients were included; 73 received TREO and 457 BU (BU ≤ 6.4 mg/kg in 134, considered RIC, BU > 6.

View Article and Find Full Text PDF

Background: Several studies have described a potential anti-tumour effect of cannabinoids (CNB). CNB receptor 2 (CB2) is mostly present in hematopoietic stem cells (HSC). The present study evaluates the anti-leukaemic effect of CNB.

View Article and Find Full Text PDF

Objective: Menopause and chronic graft-versus-host disease (cGvHD) are the leading causes of morbidity after allogeneic hematopoietic stem cell transplantation (alloHSCT). Genitalia are one of the target organs of cGvHD causing sexual dysfunction and local symptoms, which may impair women's quality of life. The aim of this study is to describe the prevalence and clinical characteristics of genital cGvHD.

View Article and Find Full Text PDF
Article Synopsis
  • Loss of B-cell aplasia (BCA) is linked to the effectiveness of CD19 CAR-T therapy for B-cell acute lymphoblastic leukemia, with most relapses occurring in the first months post-infusion, particularly in patients with high tumor burden (HTB).
  • A study of 73 patients showed that those with HTB had significantly lower event-free survival (EFS) rates (19.3%) compared to those with low tumor burden (LTB) (67.2%), and relapses were more likely to be CD19-negative in the HTB group.
  • The findings suggest that BCA loss is associated with a higher risk of CD19-positive relapses and that patients with HTB might benefit from additional
View Article and Find Full Text PDF