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Efficacy and Safety of Everolimus in Extrapancreatic Neuroendocrine Tumor: A Comprehensive Review of Literature.

BACKGROUND: Everolimus, an oral mTOR (mammalian target of rapamycin) inhibitor, is currently approved for the treatment of progressive pancr … reated with everolimus. Nine different primary sites of extrapancreatic NETs were found. The median progression-free survival ranged from 12.0 to 29.9 months. The median time to progression was not reached in a phase II prospective study, and the interval to progression ranged from 12 to 36 months in 5 clinical cases. Objective responses were observed in 7 prospective stud …

Long-Term Effects of Cyclophosphamide in Sustained Remission in Childhood Steroid-resistant Nephrotic Syndrome.

Nephrotic syndrome (NS) is a chronic renal disease that can progress into end-stage renal disease within 5 years if no remission occurs. Achieving optimal remission is important to obtain better outcomes of treating childhood steroid-resistant NS (SRNS). This study aimed to determine the survival, long-term outcomes, and factors associated with the sustained remission of SRNS patients after cyclophosphamide therapy. We retrospectively studied 33 children (24 boys) with SRNS treated with cyclophosphamide therapy over 2 years. Relapse-free survival was estimated using the Kaplan-Meier method. Th

Chromosome 3p loss of heterozygosity and reduced expression of H3K36me3 correlate with longer relapse-free survival in sacral conventional chordoma.

Conventional chordoma is a rare slow-growing malignant tumor of notochordal origin primarily arising at the base of the skull and sacrococcygeal bones. Chordoma may arise from its benign counterpart, benign notochordal cell tumors, and can also undergo dedifferentiation progressing into dedifferentiated chordoma. No study has directly compared the genomic alterations among these tumors comprising a morphologic continuum. Our prior study identified frequent chromosome 3p loss of heterozygosity and minimal deleted regions on chromosome 3 encompassing SETD2, encoding a histone methyltransferase i

Azacitidine Post-transplant Maintenance Improves Disease Progression in High-Risk Acute Myeloid Leukemia and Myelodysplastic Syndrome.

BACKGROUND: Maintenance after allogeneic hematopoietic cell transplantation (alloHCT) with hypomethylating agents has yielded conflicting re … ted to patients with high-risk AML/MDS (HR = 0.4, 95% CI = 0.2-0.8, P = .009). This led to improved progression-free survival both in high-risk AML and MDS patients with maintenance (HR = 0.2, 95% CI = 0.1-0.6, P = .004 and HR = 0.4, 95% CI = 0.2-0.9, P = .04). CONCLUSION: AZA maintenance was associated with a lower progression rate in patients with high-risk FLT3-negativ …

Prognostic significance of angiogenesis in superficial bladder cancer.

OBJECTIVE: To assess the prognostic significance of angiogenesis parameters such as microvessel density (MVD) and vascular endothelial growth factor (VEGF) in superficial bladder cancer. PATIENTS AND METHODS: We studied 127 superficial bladder cancer samples immunohistochemically for the above factors. We compared them with standard clinicopathological features (grade, stage, concurrent in situ, multifocality, primary or recurrent status) as well as with p53 expression, recurrence and progression to muscle infiltrating disease. RESULTS: During a 36 months median follow up of 109 patients wit

Re-exploration of prognosis in type B thymomas: establishment of a predictive nomogram model.

OBJECTIVE: To explore the risk factors for disease progression after initial treatment of type B thymomas using a predictive nomogram model. … nuary 2012 and December 2021. The median follow-up was 58 months (range: 1-128 months). The 10-year progression-free survival (PFS) was 91.8%. The final nomogram model included R0 resection status and Masaoka stage, with a concordance index of 0.880. Non-R0 resection and advanced Masaoka stage were negative prognostic factors for disease progression (p < 0.001). No benefit …

HPV status and favourable outcome in vulvar squamous cancer.

It is universally accepted that high-risk human papillomavirus (HR-HPV) is the cause of cervical dysplasia and cancer. More recently, it has … ncer (hazard ratio, 0.22, p = 0.001). In the adjusted analysis, HR-HPV was associated with improved progression-free survival of VSCC compared to those with HPV negative tumours (hazard ratio, 0.32, p = 0.02).

Suprasellar pilocytic astrocytoma: one national centre's experience.

INTRODUCTION: Pilocytic astrocytomas in the supratentorial compartment make up 20 % of all brain tumours in children with only 5 % of these arising in the suprasellar region. Optic pathway gliomas or suprasellar gliomas are often seen in neurofibromatosis type 1 (NF1) patients. Given their location, suprasellar pilocytic astrocytomas are challenging to manage surgically with high morbidity rates from surgical resection. We assess our cohort of patients with suprasellar pilocytic astrocytoma and document our experience. METHOD: A retrospective review of patients diagnosed with suprasellar glio

Upadacitinib Versus Tofacitinib in Anti-MDA5-Positive Dermatomyositis With Interstitial Lung Disease: A Multi-Centre Cohort Study Emulating a Target Trial.

BACKGROUND AND OBJECTIVE: The treatment of anti-melanoma differentiation-associated gene 5 antibody-positive dermatomyositis with interstitial lung disease (MDA5+DM-ILD) remains a significant clinical challenge. To compare the effectiveness and safety of upadacitinib (UPA) versus tofacitinib (TOFA) upon 6-month lung transplantation-free survival in newly diagnosed MDA5+DM-ILD patients. METHODS: This multi-centre cohort study in China enrolled MDA5+DM-ILD patients treated from January 2020 to February 2025. Prevalent/incident new users and non-inferior design along with inverse probability tre

Childhood acute lymphoblastic leukemia treatment in an academic hospital in Kenya: Treatment outcomes and health-care providers' perspectives.

BACKGROUND: Early deaths and treatment nonadherence are major reasons for low childhood acute lymphoblastic leukemia (ALL) survival in low- and middle-income countries. This study assessed treatment outcomes of children presenting with ALL and evaluated perspectives of health-care providers (HCP) on ALL treatment at a Kenyan academic hospital. METHODS: This was a combined retrospective medical records and cross-sectional questionnaire study. Treatment outcomes of 136 children diagnosed with ALL between 2010 and 2016 were collected. Questionnaires were completed by 245 HCP (response rate, 86%)

CD19 CAR T-cell Escape in Large B-cell Lymphoma: Antigen Density Matters after All.

In this issue of Blood Cancer Discovery, Corona, Gillmor, and colleagues show that quantitative CD19 density measured before chimeric antigen receptor (CAR) T-cell infusion stratifies progression-free survival in large B-cell lymphoma, yet low antigen density rarely reflects a genetic lesion at the CD19 locus itself, suggesting that other mechanisms of CD19 downregulation may promote CAR T-cell escape. Moreover, Newsam and colleagues offer one explanation-recurrent del(3p21.31)/RHOA loss that results in decreased CD19 in trans and impairs CAR T-cell efficacy. See related article by Corona, Gil…

The PROTAC milestone: FDA approval of vepdegestrant (ARV-471) for ESR1-mutated breast cancer.

The 2026 FDA approval of vepdegestrant (ARV-471), an oral proteolysis-targeting chimera (PROTAC) that targets the estrogen receptor α (ERα), represents a historic milestone for targeted protein degradation. Unlike selective estrogen receptor degraders that operate via occupancy-driven destabilization, vepdegestrant catalytically recruits the CRBN E3 ligase to ERα, inducing its ubiquitination and proteasomal degradation. In the Phase III VERITAC-2 trial, vepdegestrant significantly improved progression-free survival versus fulvestrant in ESR1-mutated, endocrine-resistant breast cancer. This mil…

Взгляды на амбулаторное лечение би-специальных T-Cell-Worker терапий для нескольких миелом.

В последние несколько лет была разработана новая перспективная терапия под названием &lt; &lt; Участок Т-клеток &gt; &gt; (TCE), которая в настоящее время доступна во многих странах для пациентов с повторной или рефрактной множественной миеломой. Лица, занимающиеся деятельностью Т-клеток, связаны с устойчивыми показателями эффективности и беспрогрессивными льготами по выживанию у пациентов с хорошо обработанной миеломой. Однако такие осложнения, как синдром высвобождения цитокина (CRS), синдром нейротоксичности, связанный с иммунным эффектом клеток (ICANS), и инфекционные заболевания затрудняю

Совпадение в аннотации оригинала
In the past few years, a new promising therapy, called bispecific T-cell engager (TCE), has been developed and is now available in many countries for patients with relapsed or refractory multiple myeloma. T-cell engagers are associated with sustained efficacy and progression-free survival benefits in patients with heavily treated myeloma. However, complicati…

Lung enteric-type adenocarcinoma with gastric metastasis: a rare case report and literature review.

Lung enteric-type adenocarcinoma (ETAC) is a rare subtype of non-small cell lung cancer (NSCLC), comprising approximately 0.6% of all primary lung adenocarcinomas. It is characterized by a tendency for early metastasis and a prognosis comparable to that of common lung adenocarcinoma. This case report described a patient with lung-ETAC who developed gastric metastasis. The patient underwent treatment with chemotherapy and a PD-1 inhibitor, resulting in disease remission with a progression-free survival (PFS) of 8 months. The follow-up time was 13 months. This case report was aimed to enhance un…

A Role for Germline Variants in Multiple Myeloma?

In Blood Cancer Discovery, Thibaud and colleagues report the incidence of pathogenic germline variants (PGV) in patients with multiple myeloma and that these PGVs are associated with DNA repair pathway genes, including BRCA1 and BRCA2. They find an association of patients with PGVs and previous family or personal history of cancer, and that these patients are diagnosed slightly earlier than those without PGVs. Patients with PGVs had a longer progression-free survival than those without PGVs when they received high-dose melphalan and autologous stem cell transplant, providing a therapeutic rati…

Single gene mutations and prognosis in limited-stage follicular lymphoma treated with radiation therapy.

Radiotherapy is routinely used for management of limited-stage follicular lymphoma (FL), yet half of patients ultimately relapse. We hypothesized that the presence of specific gene mutations may predict outcomes. We performed targeted sequencing of a 69-gene panel in 117 limited-stage FL patients treated with radiotherapy and identified recurrently mutated genes. CREBBP was most frequently mutated, and mutated CREBBP was associated with inferior progression-free survival, though not after false discovery rate adjustment. This association failed to validate in an independent cohort. We conclude…

Identifying clinical response to daratumumab therapy in relapsed/refractory multiple myeloma using a patient-derived in vitro model.

Response to daratumumab in patients with relapsed/refractory multiple myeloma is heterogeneous, and a reliable biomarker of response is lacking. We aimed to develop a method that identifies response to daratumumab therapy. Patient-derived MM cells were collected before start of daratumumab treatment and were cultured in a hydrogel-based culture system. The extent of antibody-dependent cellular cytotoxicity and complement-dependent cytotoxicity in vitro was associated with both clinical response and progression-free survival in corresponding patients. Together, our results demonstrate that in v…

[Next treatment for TKI-resistant CML].

Tyrosine kinase inhibitors (TKIs) have significantly improved the prognosis of chronic myeloid leukemia (CML) in the chronic phase. However, only 50-60% of patients stay on the same TKI for 5 years, and the long-term progression-free survival rate is significantly reduced if an early molecular response is not achieved. Possible mechanisms of therapeutic resistance against BCR::ABL1 dependent clones include point mutations in the ABL1 kinase domain, BCR::ABL1 splicing variants, BCR::ABL1 overexpression, and altered pharmacokinetics by the ABC transporter. Ponatinib, the most potent inhibitor am…

Secondary Cytoreductive Surgery With Hyperthermic Intraperitoneal Chemotherapy for Advanced or Recurrent Mucinous Ovarian Cancer.

BACKGROUND: Advanced or recurrent primary mucinous ovarian cancer is typically incurable and associated with short progression-free and overall survival when treated with standard chemotherapy. Novel approaches are desperately needed for women with this disease. CASES: Two patients with advanced or recurrent primary mucinous ovarian cancer were treated with secondary cytoreductive surgery (CRS) with hyperthermic intraperitoneal chemotherapy (HIPEC). No additional chemotherapy was administered postoperatively. Both patients achieved a complete and durable response, with no evidence of recurren

Pretreatment blast-to-lymphocyte ratio as a prognostic marker for CD19/CD3-bispecific T cell-engaging antibodies (blinatumomab) treatment against relapsed or refractory B-precursor acute lymphoblastic leukemia.

Blinatumomab is an immunotherapy drug approved for the treatment of acute lymphoblastic leukemia. Since not all patients respond to blinatum … , along with the treatment groups, each of the following was independently correlated with superior progression-free survival: salvage-treatment phase, allogeneic stem cell transplantation, and pre-treatment ratio of bone marrow blasts-to-peripheral blood lymphocytes < 25.