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Indications for haematopoietic cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2022.

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From toxicity to immunity: Evaluating CAR-T and conventional therapies in multiple myeloma through quality-adjusted life year (QALY) outcomes.

Multiple myeloma (MM) remains characterized by recurrent relapse and cumulative treatment burden despite major therapeutic advances. This study compared conventional stepwise regimens with CAR-T cell therapy in terms of QoL, toxicity, cost, and ethical considerations within a model-based comparative framework. This comparative pharmacoeconomic modeling analysis was based on published clinical trial data. Kaplan-Meier survival curves were digitized to estimate mean overall survival (OS) and progression-free survival (PFS) using area-under-the-curve integration. Treatment costs were calculated b

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… Thiotepa, Busulfan, Cyclophosphamide: Effective but Toxic Conditioning Regimen Prior to Autologous Hematopoietic Stem Cell Transplantation in Central Nervous System Lymphoma. Med Sci 2023; 11: 14. 10.3390/medsci11010014 32. İlhan Ç, Suyanı E, Sucak GT, et al. …

Polypharmacy and medication use in patients with chronic kidney disease with and without kidney replacement therapy compared to matched controls.

BackgroundThis study aims to examine polypharmacy (PP) prevalence in patients with chronic kidney disease (CKD) Stage G4/G5 and patients with kidney replacement therapy (KRT) compared with matched controls from the general population. Furthermore, we examine risk factors for PP and describe the most commonly dispensed medications.MethodsDutch health claims data were used to identify three patient groups: CKD Stage G4/G5, dialysis and kidney transplant patients. Each patient was matched to two controls based on age, sex and socio-economic status (SES) score. We differentiated between 'all medic

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… ATC code | Name L01AA01 | Cyclofosfamide L01AA02 | Chloorambucil L01AA03 | Melfalan L01AA09 | Bendamustine L01AB01 | Busulfan L01AC01 | Thiotepa L01AD02 | Lomustine L01AX03 | Temozolomide L01BA04 | Pemetrexed L01BB03 | Tioguanine L01BB05 | Fludarabine L01BB06 | Clofarabine L01BB07 | Nelarabine L01BC01 …

In vitro models of breast cancer bone metastasis: analyzing drug resistance through the lens of the microenvironment.

Even though breast cancers usually have a good outcome compared to other tumors, the cancer can progress and create metastases in different parts of the organism, the bone being a predilection locus. These metastases are usually the cause of death, as they are mostly resistant to treatments. This resistance can be caused by intrinsic properties of the tumor, such as its heterogeneity, but it can also be due to the protective role of the microenvironment. By activating signaling pathways protecting cancer cells when exposed to chemotherapy, contributing to their ability to reach dormancy, or ev

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… metastatic BC after other treatments Tecentriq | Atezolizumab | PD-L1 inhibitor | Allow T cells to kill the cancer cells by blockig the PD-1/PD-L1 interaction | Lung, liver, colon, hormone glands, cardiotoxic issues | FDA approved for metastatic TN , PD-L1+ with Abraxane Neutropenia, neuropathy Thioplex | Thiotepa …

Role of Stem Cell Transplantation in Multiple Myeloma.

Autologous stem cell transplantation (auto-SCT) has been the standard of care in eligible newly diagnosed multiple myeloma (MM) patients. Outcomes of patients with MM have improved significantly due to the advent of several novel drugs. Upfront use of these drugs in induction therapy has significantly increased the rate and depth of responses that have translated into longer remission and survival. This has now raised a debate regarding the role and relevance of auto-SCT in the management of myeloma. However, clinical trials have confirmed the utility of auto-SCT even in the era of novel drugs

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… Comparison of high-dose melphalan with a more intensive regimen of thiotepa, busulfan, and cyclophosphamide for patients with multiple myeloma. Cancer. 2004;100:2607–2612. doi: 10.1002/cncr.20294. 44. …

Interpreting clinical assays for histone deacetylase inhibitors.

As opposed to genetics, dealing with gene expressions by direct DNA sequence modifications, the term epigenetics applies to all the external influences that target the chromatin structure of cells with impact on gene expression unrelated to the sequence coding of DNA itself. In normal cells, epigenetics modulates gene expression through all development steps. When "imprinted" early by the environment, epigenetic changes influence the organism at an early stage and can be transmitted to the progeny. Together with DNA sequence alterations, DNA aberrant cytosine methylation and microRNA deregulat

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… This was different from what was seen with 5-fluorouracil (5-FU), Cisplatin, Paclitaxel, or Thiotepa. …

Long-Term Health Effects of Curative Therapies on Heart, Lungs, and Kidneys for Individuals with Sickle Cell Disease Compared to Those with Hematologic Malignancies.

The goal of curing children and adults with sickle cell disease (SCD) is to maximize benefits and minimize intermediate and long-term adverse outcomes so that individuals can live an average life span with a high quality of life. While greater than 2000 individuals with SCD have been treated with curative therapy, systematic studies have not been performed to evaluate the long-term health effects of hematopoietic stem cell transplant (HSCT) in this population. Individuals with SCD suffer progressive heart, lung, and kidney disease prior to curative therapy. In adults, these sequalae are associ

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… Haploidentical Bone Marrow Transplantation with Post-Transplantation Cyclophosphamide Plus Thiotepa Improves Donor Engraftment in Patients with Sickle Cell Anemia: Results of an International Learning Collaborative. Biol. …

Bradyarrhythmias Associated with Oncologic Treatment-An Updated Review.

Background/Objectives: Bradyarrhythmias are an under-recognized component of cancer therapy-related cardiovascular toxicity. As survivorship improves and exposure to targeted and immune therapies increases, clinicians increasingly face clinically relevant sinus node dysfunction and atrioventricular (AV) conduction disease that may interrupt otherwise effective oncologic treatment. We aimed to synthesize evidence on the incidence, phenotype, mechanisms, and management of bradyarrhythmias across major anticancer drug classes. Methods: We performed a narrative review of PubMed and major clinical

A Novel Approach to Prognostic Factors and Risk Stratification in Pediatric AML: Case Report and Literature Review.

Acute myeloid leukemia (AML), the second most common type of leukemia in children, is a heterogeneous disease known to be caused by genetic, epigenetic, and transcriptional changes, predominantly somatic and germline abnormalities. Despite significant improvement of overall survival rates, the prognosis of pediatric AML remains unfavorable in comparison with acute lymphoblastic leukemia (ALL), especially in relapsed or refractory settings. The current status and future directions are focused on establishing an accurate diagnosis and treatment strategies based on the genomic background. Next-ge

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… The patient underwent MUD peripheral blood stem cell transplant (PBSCT) CD34—7 × 10 6 /bw kg, CD3—3.5 × 10 8 /bw kg, preceded by myeloablative conditioning (fludarabine 30 mg/m 2 , days -7, -6, -5, -4, -3 + treosulfan 10 mg/m 2 , days -6, -5, -4 + thiotepa 2 × 5 mg/bw kg/day on day -2). …

Optimizing treatment efficacy and fertility preservation in patients undergoing hematopoietic stem cell transplantation: A narrative review of ovarian shielding with total-body irradiation or treosulfan-based conditioning regimens.

BackgroundPediatric and adolescent/young adult (AYA) patients with hematologic malignancies often require hematopoietic stem cell transplantation (HSCT) using conditioning regimens that pose high risks for gonadal toxicity. Traditional protocols, including total body irradiation (TBI) and busulfan-based regimens, can impair fertility. This review explores the potential of gonadal shielding during TBI and treosulfan-based conditioning as strategies to optimize treatment efficacy while preserving fertility.MethodsA PubMed search up to February 2025 was performed for English, peer-reviewed articl

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… | ESMO [5] High risk | Radiation therapy Alkylating agents (cyclophosphamide, ifosfamide, procarbazine, cisplatin, chlorambucil, carmustine, lomustine, melphalan, thiotepa, busulfan, and mechlorethamine) with CED >5 g/m 2 for germ cells and 20 g/m 2 for somatic cells Conditioning chemotherapy for bone …

Liposomes for the Treatment of Brain Cancer-A Review.

Due to their biocompatibility, non-toxicity, and surface-conjugation capabilities, liposomes are effective nanocarriers that can encapsulate chemotherapeutic drugs and facilitate targeted delivery across the blood-brain barrier (BBB). Additionally, strategies have been explored to synthesize liposomes that respond to internal and/or external stimuli to release their payload controllably. Although research into liposomes for brain cancer treatment is still in its infancy, these systems have great potential to fundamentally change the drug delivery landscape. This review paper attempts to consol

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… Drugs currently available in the commercial market to treat brain tumors include thiotepa, temozolomide, methotrexate, topotecan, irinotecan, cisplatin, and carboplatin. …

Immunotherapies for pediatric cancer: current landscape and future perspectives.

The advent of immunotherapy has revolutionized how we manage and treat cancer. While the majority of immunotherapy-related studies performed to date have focused on adult malignancies, a handful of these therapies have also recently found success within the pediatric space. In this review, we examine the immunotherapeutic agents that have achieved the approval of the US Food and Drug Administration for treating childhood cancers, highlighting their development, mechanisms of action, and the lessons learned from the seminal clinical trials that ultimately led to their approval. We also shine a

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… VITA) | III | Ewing sarcoma family of tumors | Vigil | Autologous whole cell cancer vaccine with chemotherapy | NCT03495921 | [ 96 ] Irinotecan Temozolomide Haploidentical stem cell transplantation in neuroblastoma | I | Neuroblastoma | Haploidentical stem cells, mesenchymal stem cells fludarabine, thiotepa …

The 48th Annual Meeting of the European Society for Blood and Marrow Transplantation: Nurses Group - Poster Sessions (NP001 - NP054).

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Evolution of Cancer Pharmacological Treatments at the Turn of the Third Millennium.

The medical history of cancer began millennia ago. Historical findings of patients with cancer date back to ancient Egyptian and Greek civilizations, where this disease was predominantly treated with radical surgery and cautery that were often ineffective, leading to the death of patients. Over the centuries, important discoveries allowed to identify the biological and pathological features of tumors, without however contributing to the development of effective therapeutic approaches until the end of the 1800s, when the discovery of X-rays and their use for the treatment of tumors provided the

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… Other alkylating agents are represented by nitrosourea compounds (carmustine, lomustine, semustin, streptozocin, nimustine, tallimustine, photemustine), alkyl sulfates (busulfan, treosulfan, mannosulfan), ethyleneimine derivatives (thiotepa, triazichinone), epoxides (etoglucide), triazene compounds ( …

Overview of Dual-Acting Drug Methotrexate in Different Neurological Diseases, Autoimmune Pathologies and Cancers.

Methotrexate, a structural analogue of folic acid, is one of the most effective and extensively used drugs for treating many kinds of cancer or severe and resistant forms of autoimmune diseases. In this paper, we take an overview of the present state of knowledge with regards to complex mechanisms of methotrexate action and its applications as immunosuppressive drug or chemotherapeutic agent in oncological combination therapy. In addition, the issue of the potential benefits of methotrexate in the development of neurological disorders in Alzheimer's disease or myasthenia gravis will be discuss

2020 CIS Annual Meeting: Immune Deficiency & Dysregulation North American Conference.

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International Recommendations for Screening and Preventative Practices for Long-Term Survivors of Transplantation and Cellular Therapy: A 2023 Update.

As hematopoietic cell transplantation (HCT) and cellular therapy expand to new indications and international access improves, the number of HCTs performed annually continues to rise. Parallel improvements in HCT techniques and supportive care entails more patients surviving long term, creating further emphasis on survivorship needs. Survivors are at risk for developing late complications secondary to pretransplantation, peritransplantation, and post-transplantation exposures and other underlying risk factors. Guidelines for screening and preventive practices for HCT survivors were originally p

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… Potential causes include infection or immunosuppression (with CNIs), neurotoxic chemotherapy (eg, methotrexate, cytarabine, busulfan, thiotepa) and other medications, TA-TMA, cranial radiation or TBI, radiation-induced vasculitis, underlying disease (eg, cerebrovascular disease, sickle cell disease, …

Immunosenescence: signaling pathways, diseases and therapeutic targets.

Immunosenescence refers to the abnormal activation or dysfunction of the immune system as people age. Inflammaging is a typical pathological inflammatory state associated with immunosenescence and is characterized by excessive expression of proinflammatory cytokines in aged immune cells. Chronic inflammation contributes to a variety of age-related diseases, such as neurodegenerative disease, cancer, infectious disease, and autoimmune diseases. Although not fully understood, recent studies contribute greatly to uncovering the underlying mechanisms of immunosenescence at the molecular and cellul

Therapeutic Antibodies for Myeloid Neoplasms-Current Developments and Future Directions.

Therapeutic monoclonal antibodies (mAbs) such as antibody-drug conjugates, ligand-receptor antagonists, immune checkpoint inhibitors and bispecific T cell engagers have shown impressive efficacy in the treatment of multiple human cancers. Numerous therapeutic mAbs that have been developed for myeloid neoplasms, including acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), are currently investigated in clinical trials. Because AML and MDS originate from malignantly transformed hematopoietic stem/progenitor cells-the so-called leukemic stem cells (LSCs) that are highly resistant to

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… Clinical trials identifier | Trial status ALL, AML, myeloid sarcoma, CML, JMML, MDS, NHL CD19-positive R/R after first aHSCT ≤21 years old Suitable aHSCT donor available | Blinatumomab + ATG + cyclophosphamide + fludarabine + G-CSF + melphalan + mesna + rituximab (anti-CD20) + tacrolimus/sirolimus + thiotepa …

Childhood Cancer: Occurrence, Treatment and Risk of Second Primary Malignancies.

Cancer represents the leading cause of disease-related death and treatment-associated morbidity in children with an increasing trend in recent decades worldwide. Nevertheless, the 5-year survival of childhood cancer patients has been raised impressively to more than 80% during the past decades, primarily attributed to improved diagnostic technologies and multiagent cytotoxic regimens. This strong benefit of more efficient tumor control and prolonged survival is compromised by an increased risk of adverse and fatal late sequelae. Long-term survivors of pediatric tumors are at the utmost risk fo

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… If applicable, CT regimes administered with total body EBRT before HSCT consist of different combinations of etoposide, cyclophosphamide, melphalan, vincristine, cytosine arabinoside, thiotepa, and fludarabine. …