European Journal of Haematology最新文献

Spontaneous remission (SR) in pediatric acute myeloid leukemia (AML-M5) is extremely rare and is typically characterized by short duration and high relapse risk. We report a case of AML-M5 in a child at Yunnan Children's Medical Center who achieved SR following blood transfusions and anti-infective therapy, without leukemia-specific treatment, but relapsed one month later. A comprehensive literature review was also conducted to explore factors contributing to SR in pediatric leukemia, with the aim of informing treatment protocols and improving outcomes for AML patients.

Acute myeloid leukemia (AML) in elderly patients presents a major therapeutic challenge, as many are deemed unfit for intensive chemotherapy due to age, comorbidities, or frailty. Venetoclax in combination with hypomethylating agents (HMA) has emerged as a standard-of-care for this population, yet outcomes remain heterogeneous and predictive tools are limited. In this retrospective single-center study, we analyzed 52 treatment-naïve AML patients receiving venetoclax combined with either azacitidine (n = 33) or decitabine (n = 19) at the Hematology Department of Cosenza Hospital between August 2021 and June 2025. Frailty was assessed using the Clinical Frailty Scale (CSHA CFS), with 19 patients classified as low frailty (score ≤ 3) and 33 as high frailty (score > 3). The median age was 75.3 years (range 58.2–89.2), and the cohort included 33 de novo and 19 secondary AML cases. After a median follow-up of 18 months, 34 patients (65.4%) had died: 14 due to disease progression, 12 due to treatment-related toxicity—predominantly severe infections—and 5 from unrelated causes. ROC curve analysis showed that a CFS score > 3 was associated with worse survival (AUC 0.75, 95% CI 0.61–0.89, p < 0.004), with median overall survival of 7.6 months for low-frailty patients versus 2.5 months for high-frailty patients (1-year OS 44.1% vs. 18.7%, p = 0.031). Multivariate analysis confirmed that lower frailty (p = 0.031) and azacitidine-based therapy (p = 0.025) were independently associated with improved survival. Overall response rate was 48%, including 21 complete responses (CR/CRi) and 4 partial responses. Frailty was the only significant predictor of response (p = 0.005), whereas age, sex, type of AML, ELN risk score, renal function, BMI, or type of HMA did not significantly influence outcomes. Grade 3–4 treatment-related adverse events occurred in all patients, predominantly hematological; non-hematological events included infections (61.9%), cardiotoxicity (11.9%), and liver toxicity (9.5%). High-frailty patients experienced a higher incidence of infections (72.7% vs. 36.8%, p = 0.02) and hospitalizations (57.6% vs. 21.1%, p = 0.011). These results suggest that the CSHA CFS is a simple and clinically meaningful tool to stratify elderly AML patients for venetoclax–HMA therapy, identifying those at higher risk of treatment-related complications and poor survival. Incorporating frailty assessment into routine practice may enhance patient selection, optimize supportive care, and guide individualized therapeutic decisions. Prospective, multicenter studies are warranted to validate these findings and refine the use of frailty-guided treatment strategies in this vulnerable population.

Hematopoietic stem cell transplantation (HSCT) involves the intravenous infusion of stem cells to restore bone marrow function in patients with bone marrow disorders. This study aimed to evaluate the complications of HSCT, mainly gastrointestinal, hepatobiliary, urinary, and pulmonary complications, and their incidence, severity, impacts on survival, and the importance of the surgical role in managing these complications. We retrospectively studied 433 patients treated at the IOP-GRAACC/Unifesp for a period of 10 years, with an average age of 8.8 years. The most frequent primary diseases were leukemia (33.5%) and lymphoma (13.2%). Most patients had complications (96.3%), of which half had only one complication (51.3%). Approximately 298 patients had gastrointestinal complications, with mucositis being the most common one (95.3%). Approximately 6.7% of the patients had some hepatobiliary complications; sinusoidal obstruction syndrome (SOS) occurred in 72.4% of these. Only 9.2% of patients had hemorrhagic cystitis, and most cases were classified as acute, grade I, or mild. Pulmonary complications including viral pneumonia (35.9%), bacterial pneumonia (23.1%), and fungal pneumonia (15.4%) were also recorded. Only 2.1% of patients had aspergillosis. Approximately 37.6% of the patients died, 55.2% were alive, and 7.2% were alive with the disease. The most frequent cause of death among patients was disease progression (72.4%). Only two patients underwent surgery. We concluded that the incidence of HSCT complications was 96.3%; however, it was not possible to determine the risk factors for the need for surgical treatment in these patients, although surgical evaluation in the decision-making process remains essential.

Primary cutaneous B-cell lymphomas (CBCL) represent a clinically and biologically heterogeneous group of extranodal non-Hodgkin lymphomas confined to the skin at the time of diagnosis. They account for approximately 25% of all primary cutaneous lymphomas and are subclassified into distinct entities according to the World Health Organization—European Organization of Research and Treatment of Cancer (WHO–EORTC) classification and International Consensus Classification (ICC). including primary cutaneous follicle center lymphoma (PCFCL), primary cutaneous marginal zone lymphoma (PCMZL), primary cutaneous diffuse large B-cell lymphoma, leg type (PCDLBCL-LT), intravascular large B-cell lymphoma (IVLBCL) and Epstein–Barr virus–positive mucocutaneous ulcer (EBVMCU). These subtypes differ significantly in clinical behavior, histopathological features, molecular alterations, and prognosis. Indolent forms such as PCFCL and PCMZL are typically managed with local therapies and are associated with an excellent prognosis. In contrast, aggressive variants such as PCDLBCL-LT require systemic treatment and are linked to poorer outcomes. EBVMCU, despite its alarming histological appearance, generally follows a benign and self-limiting course. This review provides an updated overview of the current diagnostic criteria, clinical management strategies, and emerging molecular insights for each CBCL subtype. It also emphasizes the importance of a multidisciplinary approach and discusses the challenges of prognostication, along with the evolving but still limited role of innovative therapies.

Hematologic malignancies represent the most common cancers in children. While the mainstays of treatment are chemotherapy and potentially hematopoietic stem cell transplant, minimally invasive surgery (MIS) has a role in the diagnosis and management of complications related both to disease and therapy as well as common pediatric surgical conditions. MIS can be considered in the management of adverse events of disease and therapy such as pneumatosis, bowel perforation, or pneumoperitoneum in stable patients as a first-look alternative to open surgery. Additionally, given the extent of therapy for many children, common surgical problems such as the need for enteral access, appendicitis, and cholecystitis can occur, and laparoscopic options should be considered for management to enable a faster return to chemotherapy. Finally, MIS can assist with diagnosis through lymph node or tissue biopsy. The utilization of MIS in pediatric patients with hematologic malignancies is a safe and feasible approach for a variety of surgical problems. Nevertheless, current evidence is largely limited to retrospective series and case reports, and prospective multicenter studies will be required to validate these approaches and emerging technologies such as augmented reality and robotics.

Previous studies investigating the role of TP53 mutations in chronic phase MPN have yielded inconsistent results. As such, the clinical relevance of these mutations remains to be elucidated. We report a case of a 67-year-old woman with a leukemic transformation of a post-polycythaemia vera myelofibrosis (post-PV MF) that culminated in the rare development of a myeloid sarcoma. During a 4-year follow-up, the patient had a stable JAK2 Val617Phe and NFE2 mutation (Variant Allele Frequencies (VAF's) 85%–87% and 43%–50%, respectively) and low-burden TP53 mutation (VAF 2%–7%) in blood and bone marrow. Despite having a low-risk post-PV MF, the patient soon presented with an aggressive lytic lesion in the humerus, which proved to be a myeloid sarcoma. The sarcoma was positive for JAK2 Val617Phe but, interestingly, was also highly enriched for the TP53 mutation (VAF 81%), implicating a role of the TP53 mutation in the leukemic progression. This case provides molecular evidence that a TP53 mutation, even at a low burden, may contribute to leukemic progression of a chronic phase myeloproliferative neoplasm (MPN). These observations increase our understanding of the pathophysiologic behavior of TP53 mutations and underscore the importance of further causal research towards the clinical implications of these mutations in chronic phase MPN.