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1.
Front Immunol ; 15: 1384640, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-38720904

RESUMO

Background: For children with severe aplastic anemia, if the first immunosuppressive therapy (IST) fails, it is not recommended to choose a second IST. Therefore, for patients without matched sibling donor (MSD) and matched unrelated donor (MUD), haploidentical hematopoietic stem cell transplantation (Haplo-HSCT) can be chosen as a salvage treatment. This article aims to explore the comparison between upfront Haplo-HSCT and salvage Haplo-HSCT after IST. Methods: 29 patients received salvage Haplo-HSCT, and 50 patients received upfront Haplo-HSCT. The two groups received Bu (Busulfan, 3.2mg/kg/d*2d on days -9 to-8), CY (Cyclophosphamide, 60mg/kg/d*2d on days -4 to-3), Flu (fludarabine, 40mg/m2/d*5d on days -9 to -5) and rabbit ATG (Anti-thymocyte globulin, total dose 10mg/kg divided into days -4 to -2). Results: The OS of the salvage Haplo-HSCT group showed no difference to the upfront Haplo-HSCT group (80.2 ± 8.0% vs. 88.7 ± 4.8%, p=0.37). The FFS of the salvage Haplo-HSCT group also showed no difference to the frontline Haplo-HSCT group (75 ± 8.2% vs. 84.9 ± 5.3%, p=0.27). There was no significant difference in the incidence of other complications after transplantation between the two groups, except for thrombotic microangiopathy (TMA). In the grouping analysis by graft source, the incidence of II-IV aGVHD in patients using PBSC ± BM+UCB was lower than that in the PBSC ± BM group (p=0.010). Conclusion: Upfront Haplo-HSCT and salvage Haplo-HSCT after IST in children with acquired severe aplastic anemia have similar survival outcomes. However, the risk of TMA increases after salvage Haplo-HSCT. This article provides some reference value for the treatment selection of patients. In addition, co-transplantation of umbilical cord blood may reduce the incidence of GVHD.


Assuntos
Anemia Aplástica , Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Terapia de Salvação , Transplante Haploidêntico , Humanos , Anemia Aplástica/terapia , Anemia Aplástica/mortalidade , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/métodos , Masculino , Feminino , Criança , Pré-Escolar , Terapia de Salvação/métodos , Adolescente , Doença Enxerto-Hospedeiro/etiologia , Doença Enxerto-Hospedeiro/prevenção & controle , Imunossupressores/uso terapêutico , Condicionamento Pré-Transplante/métodos , Lactente , Resultado do Tratamento , Terapia de Imunossupressão/métodos
2.
Am Soc Clin Oncol Educ Book ; 44(3): e433502, 2024 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-38728605

RESUMO

Combination chemotherapy with or without radiation has served as the primary therapeutic option for classic Hodgkin lymphoma (cHL), leading to durable remission in a majority of patients with early- and advanced-stage cHL. Patients with relapsed/refractory (RR) cHL could still be cured with salvage chemotherapy and autologous stem-cell transplantation. Brentuximab vedotin (BV) and the anti-PD-1-blocking antibodies, nivolumab and pembrolizumab, are highly effective treatments for cHL and have revolutionized the management of the disease. Recent studies incorporating BV and PD-1 blockade into salvage therapy for RR cHL and into frontline treatment regimens have changed the cHL treatment paradigm. The novel agents are also useful in the treatment of older patients who have poor outcomes with traditional therapy. This manuscript will review current strategies for approaching the management of previously untreated, RR, and challenging populations with cHL, including how to incorporate the novel agents.


Assuntos
Doença de Hodgkin , Doença de Hodgkin/terapia , Doença de Hodgkin/tratamento farmacológico , Humanos , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Recidiva Local de Neoplasia , Terapia Combinada , Terapia de Salvação/métodos , Resultado do Tratamento , Inibidores de Checkpoint Imunológico/uso terapêutico , Gerenciamento Clínico , Recidiva
4.
Rev Med Suisse ; 20(874): 978-983, 2024 May 15.
Artigo em Francês | MEDLINE | ID: mdl-38756035

RESUMO

Spatially fractionated radiotherapy is a new concept involving partial irradiation of tumor volumes. Different techniques are described: mini-beam and micro-beam radiotherapy (pre-clinical) and LATTICE radiotherapy (L-RT) (clinical). Although L-RT is emergent in clinical practice and its evidence is still limited, it has still revealed excellent outcomes. At least three clinical situations can be discussed: definitive palliative radiotherapy, dose escalation (boost) or salvage radiotherapy. The interaction between L-RT and the immune system is still under investigation. Preclinical observations have already demonstrated a strong interaction, with tumor response dependent on immune system stimulation and the generation of an abscopal effect.


La radiothérapie fractionnée dans l'espace est un nouveau concept consistant en une irradiation partielle des volumes tumoraux. Plusieurs techniques sont ainsi décrites : les radiothérapies mini-beam et micro-beam (pré-clinique) et la radiothérapie LATTICE (L-RT) (clinique). Bien que la L-RT soit relativement nouvelle dans la pratique clinique et que les preuves quant à son utilisation soient encore limitées, elle montre des résultats prometteurs. Au moins trois situations cliniques peuvent être examinées en détail : la radiothérapie palliative définitive, l'escalade de dose (boost) ou encore la radiothérapie de sauvetage. L'interaction entre la L-RT et le système immunitaire est encore en cours d'investigation, mais des observations précliniques ont déjà démontré une interaction forte, avec notamment la dépendance de la réponse tumorale à la stimulation du système immunitaire et la génération d'un effet abscopal.


Assuntos
Fracionamento da Dose de Radiação , Neoplasias , Humanos , Neoplasias/radioterapia , Cuidados Paliativos/métodos , Terapia de Salvação/métodos
5.
Clin Transplant ; 38(5): e15366, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38775798

RESUMO

In children with high-risk childhood acute leukemia who undergo allogeneic hematopoietic stem-cell transplantation (allo-HSCT), relapse is still the leading cause of treatment failure. The prognosis is poor, yet prospective studies have only limited data on risk factors and outcomes. We aimed to understand the outcomes and prognostic factors for patients with acute lymphoblastic leukemia (ALL) who relapsed following allo-HSCT. We analyzed retrospectively 46 children with childhood acute lymphoblastic leukemia who had relapsed after receiving their first alloHSCT. All these patients received salvage chemotherapy which consisted of fludarabine, cytarabine, and idarubicin before performing a second alloHSCT. The median follow-up of the 46 patients after the first transplantation was 366 days. The median time from first allo-HSCT to relapse was 278.4 ± 238.4 days. Forty-six patients received salvage chemotherapy before the second alloHSCT, and CR was achieved in 32 of 46 patients. However, only 17 (37%) of 46 patients received a second allo-HSCT, and 15 of 46 patients died from disease progression, infections, and bleeding. Twelve patients are still alive after the second allo-HSCT. Two-year overall survival (OS) was 38.9%. Local therapy was given to 10 (21.8%) patients, either as part of systemic therapy or alone. In multivariate analyses, the time of relapse and curative salvage therapy with a second allo-HSCT were identified as significant prognostic factors for OS. Children with leukemia who had relapsed after the first allo-HSCT received salvage chemotherapy. Our statistical analysis showed that the second HSCT could be beneficial for outcomes if patients relapsed beyond 180 days of the first allo-HSCT.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Leucemia-Linfoma Linfoblástico de Células Precursoras , Transplante Homólogo , Humanos , Feminino , Masculino , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Leucemia-Linfoma Linfoblástico de Células Precursoras/mortalidade , Criança , Estudos Retrospectivos , Pré-Escolar , Prognóstico , Seguimentos , Adolescente , Taxa de Sobrevida , Recidiva Local de Neoplasia/patologia , Recidiva Local de Neoplasia/terapia , Fatores de Risco , Lactente , Doença Enxerto-Hospedeiro/etiologia , Terapia de Salvação , Condicionamento Pré-Transplante , Recidiva
6.
Turk Neurosurg ; 34(3): 423-428, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-38650557

RESUMO

AIM: To evaluate the efficacy of percutaneous ventriculoatrial shunting as a salvage method for pediatric patients with abdominal complications. MATERIAL AND METHODS: Data obtained from 9 patients with ventriculoperitoneal shunt dysfunctions owing to abdominal complications, who underwent ventriculoatrial shunting as salvage treatment at a single institution between January 2019 and September 2021 were retrospectively analyzed. All operations were conducted under the guidance of intraoperative fluoroscopy and ultrasound. RESULTS: The mean age of the enrolled patients was 8.1 ± 1.2 years (2-15 years). Six (67%) patients were male and 3 (33%) were female. The mean number of the patients? ventriculoperitoneal shunt revisions until atrial catheter placement was 7.5 times. The reasons for intraperitoneal catheter failure included peritoneal adhesions in 4 (44.5%) patients, pseudocyst formation in 3 (33.3%), and peritonitis in 2 (22.2%). Seven patients from the study cohort had no problem after ventriculoatrial shunt placement. Only 1 patient had shunt dysfunction related to the ventricular catheter, and ventricular catheter and shunt valve revision was performed 26 months after ventriculoatrial shunt placement. The atrial catheter of the patient was intact. One patient died from the progression of her primary disease (medulloblastoma in the 4 < sup > th < /sup > ventricle), which was unrelated to the ventriculoatrial shunt. CONCLUSION: Percutaneous ventriculoatrial shunting under the guidance of intraoperative fluoroscopy and ultrasound is a safe, effective, and easy alternative in patients with peritoneal complications and a history of multiple operations.


Assuntos
Hidrocefalia , Derivação Ventriculoperitoneal , Humanos , Feminino , Hidrocefalia/cirurgia , Hidrocefalia/etiologia , Criança , Masculino , Pré-Escolar , Adolescente , Derivação Ventriculoperitoneal/métodos , Estudos Retrospectivos , Terapia de Salvação/métodos , Resultado do Tratamento , Complicações Pós-Operatórias/etiologia , Reoperação/estatística & dados numéricos
7.
Liver Int ; 44(6): 1464-1473, 2024 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-38581233

RESUMO

INTRODUCTION: We aim to assess the long-term outcomes of percutaneous multi-bipolar radiofrequency (mbpRFA) as the first treatment for hepatocellular carcinoma (HCC) in transplant-eligible cirrhotic patients, followed by salvage transplantation for intrahepatic distant tumour recurrence or liver failure. MATERIALS AND METHODS: We included transplant-eligible patients with cirrhosis and a first diagnosis of HCC within Milan criteria treated by upfront mbp RFA. Transplantability was defined by age <70 years, social support, absence of significant comorbidities, no active alcohol use and no recent extrahepatic cancer. Baseline variables were correlated with outcomes using the Kaplan-Meier and Cox models. RESULTS: Among 435 patients with HCC, 172 were considered as transplantable with HCCs >2 cm (53%), uninodular (87%) and AFP >100 ng/mL (13%). Median overall survival was 87 months, with 75% of patients alive at 3 years, 61% at 5 years and 43% at 10 years. Age (p = .003) and MELD>10 (p = .01) were associated with the risk of death. Recurrence occurred in 118 patients within Milan criteria in 81% of cases. Local recurrence was observed in 24.5% of cases at 10 years and distant recurrence rates were observed in 69% at 10 years. After local recurrence, 69% of patients were still alive at 10 years. At the first tumour recurrence, 75 patients (65%) were considered transplantable. Forty-one patients underwent transplantation, mainly for distant intrahepatic tumour recurrence. The overall 5-year survival post-transplantation was 72%, with a tumour recurrence of 2.4%. CONCLUSION: Upfront multi-bipolar RFA for a first diagnosis of early HCC on cirrhosis coupled with salvage liver transplantation had a favourable intention-to-treat long-term prognosis, allowing for spare grafts.


Assuntos
Carcinoma Hepatocelular , Cirrose Hepática , Neoplasias Hepáticas , Transplante de Fígado , Recidiva Local de Neoplasia , Ablação por Radiofrequência , Terapia de Salvação , Humanos , Carcinoma Hepatocelular/cirurgia , Carcinoma Hepatocelular/mortalidade , Neoplasias Hepáticas/cirurgia , Neoplasias Hepáticas/mortalidade , Masculino , Feminino , Pessoa de Meia-Idade , Terapia de Salvação/métodos , Cirrose Hepática/complicações , Cirrose Hepática/cirurgia , Idoso , Ablação por Radiofrequência/métodos , Estudos Retrospectivos , Estimativa de Kaplan-Meier , Modelos de Riscos Proporcionais , Resultado do Tratamento
8.
J Clin Neurosci ; 124: 102-108, 2024 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-38685181

RESUMO

OBJECTIVE: Parasagittal meningiomas (PM) are treated with primary microsurgery, radiosurgery (SRS), or surgery with adjuvant radiation. We investigated predictors of tumor progression requiring salvage surgery or radiation treatment. We sought to determine whether primary treatment modality, or radiologic, histologic, and clinical variables were associated with tumor progression requiring salvage treatment. METHODS: Retrospective study of 109 consecutive patients with PMs treated with primary surgery, radiation (RT), or surgery plus adjuvant RT (2000-2017) and minimum 5 years follow-up. Patient, radiologic, histologic, and treatment data were analyzed using standard statistical methods. RESULTS: Median follow up was 8.5 years. Primary treatment for PM was surgery in 76 patients, radiation in 16 patients, and surgery plus adjuvant radiation in 17 patients. Forty percent of parasagittal meningiomas in our cohort required some form of salvage treatment. On univariate analysis, brain invasion (OR: 6.93, p < 0.01), WHO grade 2/3 (OR: 4.54, p < 0.01), peritumoral edema (OR: 2.81, p = 0.01), sagittal sinus invasion (OR: 6.36, p < 0.01), sagittal sinus occlusion (OR: 4.86, p < 0.01), and non-spherical shape (OR: 3.89, p < 0.01) were significantly associated with receiving salvage treatment. On multivariate analysis, superior sagittal sinus invasion (OR: 8.22, p = 0.01) and WHO grade 2&3 (OR: 7.58, p < 0.01) were independently associated with receiving salvage treatment. There was no difference in time to salvage therapy (p = 0.11) or time to progression (p = 0.43) between patients receiving primary surgery alone, RT alone, or surgery plus adjuvant RT. Patients who had initial surgery were more likely to have peritumoral edema on preoperative imaging (p = 0.01). Median tumor volume was 19.0 cm3 in patients receiving primary surgery, 5.3 cm3 for RT, and 24.4 cm3 for surgery plus adjuvant RT (p < 0.01). CONCLUSION: Superior sagittal sinus invasion and WHO grade 2/3 are independently associated with PM progression requiring salvage therapy regardless of extent of resection or primary treatment modality. Parasagittal meningiomas have a high rate of recurrence with 80.0% of patients with WHO grade 2/3 tumors with sinus invasion requiring salvage treatment whereas only 13.6% of the WHO grade 1 tumors without sinus invasion required salvage treatment. This information is useful when counseling patients about disease management and setting expectations.


Assuntos
Neoplasias Meníngeas , Meningioma , Radiocirurgia , Terapia de Salvação , Humanos , Terapia de Salvação/métodos , Meningioma/radioterapia , Meningioma/cirurgia , Masculino , Feminino , Radiocirurgia/métodos , Pessoa de Meia-Idade , Estudos Retrospectivos , Neoplasias Meníngeas/radioterapia , Neoplasias Meníngeas/cirurgia , Idoso , Adulto , Radioterapia Adjuvante , Idoso de 80 Anos ou mais , Procedimentos Neurocirúrgicos/métodos , Seguimentos , Progressão da Doença
9.
Sci Rep ; 14(1): 9148, 2024 04 21.
Artigo em Inglês | MEDLINE | ID: mdl-38644367

RESUMO

Cryotherapy is an ablative therapy that can be used to treat localized prostate cancer. In case of recurrence, treatment options are not well-defined, and their outcomes are unknown. We therefore collected all patients treated with radiotherapy after cryotherapy for prostate cancer recurrence in Nantes (France) between 2012 and 2019. We identified ten patients. After a median follow-up of 5 years, two patients presented late grade 3 toxicities; one patient presented a grade 3 rectal hemorrhage, and one had a grade 3 hematuria. Two patients relapsed at 61 and 62 months, and three patients died of other causes. Radiotherapy to treat local prostate cancer recurrence after cryotherapy seems feasible and effective in local control. These results do not allow us to recommend this technique in current practice but are encouraging for the conduct of prospective trials.


Assuntos
Crioterapia , Recidiva Local de Neoplasia , Neoplasias da Próstata , Radioterapia de Intensidade Modulada , Terapia de Salvação , Humanos , Masculino , Neoplasias da Próstata/radioterapia , Idoso , Terapia de Salvação/métodos , Crioterapia/métodos , Radioterapia de Intensidade Modulada/métodos , Radioterapia de Intensidade Modulada/efeitos adversos , Pessoa de Meia-Idade , Recidiva Local de Neoplasia/radioterapia , Idoso de 80 Anos ou mais , Falha de Tratamento
10.
Surg Innov ; 31(3): 263-273, 2024 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-38571331

RESUMO

OBJECTIVE: We propose a pedicled perforator flap technique for salvage nipple reconstruction after initial nipple reconstruction fails in breast cancer patients. METHODS: This is a pilot study. A total of 21 female breast cancer patients who underwent nipple reconstruction following initial nipple reconstruction fails were enrolled, and salvage nipple reconstruction based pedicled perforator flap were performed between 2016 and 2020. Operative time, perforator design, postoperative complications, follow-up duration, projection of nipple, as well as patient-reported outcomes measured by the BREAST-Q and visual analogue scale (VAS) were assessed. RESULTS: Sixteen patients underwent fifth lateral intercostal artery perforator reconstruction, while 5 patients underwent fifth anterior intercostal artery perforator flap reconstruction. The surgeries were successful without intraoperative complications, with a mean operative time of 67 minutes. Postoperative complications were absent. The mean follow-up duration was 18 months. The mean nipple projection was 8 mm (range, 6-10 mm) with a shrinkage of 20% at 6 months after surgery. The average scores for psychosocial well-being, satisfaction with breasts, and satisfaction with nipples domains of the BREAST-Q significantly increased (P < .01) at 6 months post-reconstruction. Sexual well-being subdomain showed no statistical difference (P = .9369). The VAS scores for cosmesis and patient satisfaction with surgery were 9 and 9.3, respectively. CONCLUSION: The pedicled perforator flap technique for salvage nipple reconstruction is a safe and effective approach.


Assuntos
Neoplasias da Mama , Mamoplastia , Mamilos , Retalho Perfurante , Humanos , Feminino , Retalho Perfurante/irrigação sanguínea , Projetos Piloto , Neoplasias da Mama/cirurgia , Mamoplastia/métodos , Pessoa de Meia-Idade , Mamilos/cirurgia , Adulto , Satisfação do Paciente , Resultado do Tratamento , Idoso , Terapia de Salvação/métodos
11.
Lancet Haematol ; 11(5): e324-e335, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38583455

RESUMO

BACKGROUND: Whether high-dose cytarabine-based salvage chemotherapy, administered to induce complete remission in patients with poor responsive or relapsed acute myeloid leukaemia scheduled for allogeneic haematopoietic stem-cell transplantation (HSCT) after intensive conditioning confers a survival advantage, is unclear. METHODS: To test salvage chemotherapy before allogeneic HSCT, patients aged between 18 and 75 years with non-favourable-risk acute myeloid leukaemia not in complete remission after first induction or untreated first relapse were randomly assigned 1:1 to remission induction with high-dose cytarabine (3 g/m2 intravenously, 1 g/m2 intravenously for patients >60 years or with a substantial comorbidity) twice daily on days 1-3 plus mitoxantrone (10 mg/m2 intravenously) on days 3-5 or immediate allogeneic HSCT for the disease control group. Block randomisation with variable block lengths was used and patients were stratified by age, acute myeloid leukaemia risk, and disease status. The study was open label. The primary endpoint was treatment success, defined as complete remission on day 56 after allogeneic HSCT, with the aim to show non-inferiority for disease control compared with remission induction with a non-inferiority-margin of 5% and one-sided type 1 error of 2·5%. The primary endpoint was analysed in both the intention-to-treat (ITT) population and in the per-protocol population. The trial is completed and was registered at ClinicalTrials.gov, NCT02461537. FINDINGS: 281 patients were enrolled between Sept 17, 2015, and Jan 12, 2022. Of 140 patients randomly assigned to disease control, 135 (96%) proceeded to allogeneic HSCT, 97 (69%) after watchful waiting only. Of 141 patients randomly assigned to remission induction, 134 (95%) received salvage chemotherapy and 128 (91%) patients subsequently proceeded to allogeneic HSCT. In the ITT population, treatment success was observed in 116 (83%) of 140 patients in the disease control group versus 112 (79%) of 141 patients with remission induction (test for non-inferiority, p=0·036). Among per-protocol treated patients, treatment success was observed in 116 (84%) of 138 patients with disease control versus 109 (81%) of 134 patients in the remission induction group (test for non-inferiority, p=0·047). The difference in treatment success between disease control and remission induction was estimated as 3·4% (95% CI -5·8 to 12·6) for the ITT population and 2·7% (-6·3 to 11·8) for the per-protocol population. Fewer patients with disease control compared with remission induction had non-haematological adverse events grade 3 or worse (30 [21%] of 140 patients vs 86 [61%] of 141 patients, χ2 test p<0·0001). Between randomisation and the start of conditioning, with disease control two patients died from progressive acute myeloid leukaemia and zero from treatment-related complications, and with remission induction two patients died from progressive acute myeloid leukaemia and two from treatment-related complications. Between randomisation and allogeneic HSCT, patients with disease control spent a median of 27 days less in hospital than those with remission induction, ie, the median time in hospital was 15 days (range 7-64) versus 42 days (27-121, U test p<0·0001), respectively. INTERPRETATION: Non-inferiority of disease control could not be shown at the 2·5% significance level. The rate of treatment success was also not statistically better for patients with remission induction. Watchful waiting and immediate transplantation could be an alternative for fit patients with poor response or relapsed acute myeloid leukaemia who have a stem cell donor available. More randomised controlled intention-to-transplant trials are needed to define the optimal treatment before transplantation for patients with active acute myeloid leukaemia. FUNDING: DKMS and the Gert and Susanna Mayer Stiftung Foundation.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Leucemia Mieloide Aguda , Indução de Remissão , Transplante Homólogo , Humanos , Transplante de Células-Tronco Hematopoéticas/métodos , Leucemia Mieloide Aguda/terapia , Leucemia Mieloide Aguda/tratamento farmacológico , Pessoa de Meia-Idade , Masculino , Feminino , Adulto , Idoso , Citarabina/uso terapêutico , Citarabina/administração & dosagem , Adulto Jovem , Adolescente , Mitoxantrona/uso terapêutico , Mitoxantrona/administração & dosagem , Terapia de Salvação/métodos , Resultado do Tratamento , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Recidiva
13.
Zhonghua Er Ke Za Zhi ; 62(5): 467-472, 2024 May 02.
Artigo em Chinês | MEDLINE | ID: mdl-38623016

RESUMO

Objective: To analyze the efficacy and safety of the L-DEP regimen (asparaginase, liposome doxorubicin, etoposide and methylprednisolone) as a salvage therapy for the refractory primary hemophagocytic lymphohistocytosis triggered by Epstein-Barr virus infection (EBV-pHLH) in children. Methods: In this retrospective case study, clinical and laboratory data before and after L-DEP regimen of 4 children diagnosed with EBV-pHLH in Beijing Children's hospital between January 2016 and June 2022 were collected, and the efficacy and safety of L-DEP regimen for the treatment of EBV-pHLH were analyzed. Results: Among 4 patients, there were 3 females and 1 male with the age ranged from 0.8 to 7.0 years. Two of them showed compound heterozygous mutations of PRF1, one with a heterozygous mutation of UNC13D, one homozygous mutation of ITK. Before the L-DEP therapy, all of them had anemia and a soaring level of soluble CD25, 3 patients had neutropenia and thrombopenia, 3 patients had a high level of ferritin, 3 patients had hypofibrinogenemia and 1 patient had hypertriglyceridemia. After receiving 1 or 2 cycles of L-DEP treatment, three achieved remission, including complete remission (1 case) and partial remission (2 cases), and the other one had no remission. The levels of blood cell counts, soluble CD25, triglyceride, fibrinogen and albumin were recovered gradually in 3 patients who got remission. All four patients underwent hematopoietic stem cell transplantation (HSCT) after L-DEP regimen, and three survived. All patients had no severe chemotherapy related complications. The main side effects were bone marrow suppression, infection and pancreatitis, which recovered after appropriate treatments, apart from one who died from severe infection after urgent HSCT. Conclusion: L-DEP regimen could be served as an effective and safe salvage treatment for refractory pediatric EBV-pHLH, and also provide an opportunity for patients to receive HSCT.


Assuntos
Asparaginase , Infecções por Vírus Epstein-Barr , Etoposídeo , Linfo-Histiocitose Hemofagocítica , Terapia de Salvação , Humanos , Linfo-Histiocitose Hemofagocítica/terapia , Linfo-Histiocitose Hemofagocítica/tratamento farmacológico , Masculino , Feminino , Infecções por Vírus Epstein-Barr/tratamento farmacológico , Infecções por Vírus Epstein-Barr/complicações , Estudos Retrospectivos , Terapia de Salvação/métodos , Criança , Lactente , Pré-Escolar , Etoposídeo/administração & dosagem , Asparaginase/administração & dosagem , Doxorrubicina/administração & dosagem , Metilprednisolona/administração & dosagem , Mutação , Proteínas de Membrana/genética , Resultado do Tratamento , Perforina/genética , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Lipossomos , Herpesvirus Humano 4/genética
14.
BMC Cancer ; 24(1): 481, 2024 Apr 16.
Artigo em Inglês | MEDLINE | ID: mdl-38627695

RESUMO

BACKGROUND: The treatment for lung oligometastasis from colorectal cancer (CRC) remains challenging. This retrospective study aimed to compare the local tumor control, survival and procedure-related complications in CRC patients undergoing low-dose rate stereotactic ablative brachytherapy (L-SABT) versus percutaneous microwave ablation (MWA) for lung oligometastasis. METHODS: Patients between November 2017 and December 2020 were retrospectively analyzed. Local tumor progression-free survival (LTPFS) and overall survival (OS) were analyzed in the entire cohort as well as by stratified analysis based on the minimal ablation margin (MAM) around the tumor. RESULTS: The final analysis included 122 patients: 74 and 48 in the brachytherapy and MWA groups, respectively, with a median follow-up of 30.5 and 35.3 months. The 1- and 3-year LTPFS rate was 54.1% and 40.5% in the brachytherapy group versus 58.3% and 41.7% in the MWA group (P = 0.524 and 0.889, respectively). The 1- and 3-year OS rate was 75.7% and 48.6% versus 75.0% and 50.0% (P = 0.775 and 0.918, respectively). Neither LTPFS nor OS differed significantly between the patients with MAM of 5-10 mm versus > 10 mm. Pulmonary complication rate did not differ in the overall analysis, but was significantly higher in the MWA group in the subgroup analysis that only included patients with lesion within 10 mm from the key structures (P = 0.005). The increased complications was primarily bronchopleural fistula. CONCLUSIONS: Considering the caveats associated with radioisotope use in L-SABT, MWA is generally preferable. In patients with lesion within 10 mm from the key pulmonary structures, however, L-SABT could be considered as an alternative due to lower risk of bronchopleural fistula.


Assuntos
Braquiterapia , Ablação por Cateter , Neoplasias Colorretais , Fístula , Neoplasias Hepáticas , Neoplasias Pulmonares , Humanos , Neoplasias Pulmonares/radioterapia , Neoplasias Pulmonares/cirurgia , Estudos Retrospectivos , Terapia de Salvação , Micro-Ondas/efeitos adversos , Braquiterapia/efeitos adversos , Resultado do Tratamento , Pulmão/patologia , Neoplasias Colorretais/radioterapia , Neoplasias Colorretais/cirurgia , Fístula/cirurgia , Neoplasias Hepáticas/cirurgia
15.
Br J Haematol ; 204(5): 1590-1592, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38563345

RESUMO

Chimeric antigen receptor T-cell (CAR-T) therapy for the treatment of multiple myeloma (MM) has fundamentally changed the relapsed and refractory therapeutic landscape, but the disease remains incurable. Two CAR-T products, idecabtagene vicleucel (ide-cel; Abecma) and ciltacabtagene autoleucel (cilta-cel, Carvykti), have been FDA- and EMA-approved for the treatment of relapsed/refractory MM (RRMM); both target B-cell maturation antigen (BCMA), a surface glycoprotein highly expressed on MM cells. Despite deep and durable responses following CAR-T therapy, most patients will need subsequent treatment, and the optimal next-line therapy is presently unclear. Commentary on: Liu et al. Outcomes in patients with multiple myeloma receiving salvage treatment after BCMA-specific CAR-T therapy: A retrospective analysis of LEGEND-2. Br J Haematol 2024;204:1780-1789.


Assuntos
Imunoterapia Adotiva , Mieloma Múltiplo , Terapia de Salvação , Humanos , Mieloma Múltiplo/terapia , Imunoterapia Adotiva/métodos , Terapia de Salvação/métodos , Antígeno de Maturação de Linfócitos B , Receptores de Antígenos Quiméricos/uso terapêutico
16.
Anticancer Res ; 44(5): 2205-2210, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38677723

RESUMO

BACKGROUND/AIM: To evaluate the clinical outcome in men with recurrent prostate cancer (PCa) treated by salvage radiotherapy (sRT) prostate-specific membrane antigen positron emission tomography/computed tomography (PSMA PET/CT)-guided. PATIENTS AND METHODS: From January 2021 to January 2023, 33 patients who previously underwent definitive/systemic therapy were submitted to sRT PSMA PET/CT-guided for PCa recurrence: 16 (48.5%) on the prostate bed (PB), 12 (36.4%) on the lymph node (LN) and five (15.1%) on the bone. The median PSA value was 3.3 ng/ml (range=0.3-15.5 ng/ml): 0.2-0.5 ng/ml (18.2% cases), 0.51-1 ng/ml (39.4% cases) and >1 ng/ml (42.4% cases). Median 18F PSMA PET/CT standardized uptake value (SUVmax) was evaluated on PB, vs. LN vs. bones PCa recurrences and was equal to 12.5 vs. 19.0 vs. 30.1, respectively. RESULTS: Overall, at a median follow up of 12 months, 23/33 patients (69.7%) had local control without distant progression (PSA and SUVmax evaluation): 14/16 (87.5%) vs. 7/12 (58.3%) vs. 2/5 (40%) underwent sRT on the PB vs. LN vs. bone metastases, respectively. CONCLUSION: PSMA PET/CT allows to perform sRT early in men with PCa recurrence and low PSA values obtaining a complete clinical response in approximately 70% of the cases one year from treatment.


Assuntos
Recidiva Local de Neoplasia , Tomografia por Emissão de Pósitrons combinada à Tomografia Computadorizada , Antígeno Prostático Específico , Neoplasias da Próstata , Terapia de Salvação , Humanos , Masculino , Tomografia por Emissão de Pósitrons combinada à Tomografia Computadorizada/métodos , Neoplasias da Próstata/radioterapia , Neoplasias da Próstata/diagnóstico por imagem , Neoplasias da Próstata/patologia , Neoplasias da Próstata/sangue , Idoso , Antígeno Prostático Específico/sangue , Pessoa de Meia-Idade , Recidiva Local de Neoplasia/radioterapia , Recidiva Local de Neoplasia/diagnóstico por imagem , Recidiva Local de Neoplasia/patologia , Idoso de 80 Anos ou mais , Glutamato Carboxipeptidase II/metabolismo , Antígenos de Superfície , Radioterapia Guiada por Imagem/métodos
17.
J Cancer Res Ther ; 20(1): 93-97, 2024 Jan 01.
Artigo em Inglês | MEDLINE | ID: mdl-38554304

RESUMO

BACKGROUND: Treatment options for patients with metastatic sarcoma are limited. The goal of this study was to investigate the effectiveness of temozolomide in pretreated patients with soft tissue sarcoma. METHODS: We recorded the pathological, clinical, and treatment data of the patients with metastatic soft tissue sarcoma retrospectively. We evaluated the efficacy and side effects of temozolomide in this patient group. RESULTS: This study involved 16 patients. The average age was detected as 48 (21-73) years. Six (37.5%) patients had de-novo metastatic disease at diagnosis. Primary of tumors had originated from intra-abdominal (43.7%), extremity (31.3%), head-and-neck (12.5%), and intrathoracic (12.5%) regions. The patients previously had received at least two different chemotherapy regimens (75%), pazopanib (50%) and palliative radiotherapy (31.3%). Temozolomide-related median progression-free survival time was found as 3.5 (95% CI, 2.6-4.3) months. One patient (6.3%) had a partial response, while four patients (25%) had stable disease. Nine individuals (56.3%) had grade 1-2 adverse events, while one patient (6.3%) had grade 3-4 adverse events. CONCLUSIONS: We observed that temozolomide was well tolerated but had limited efficacy in the treatment of metastatic sarcoma patients. In patients with extensively pretreated soft tissue sarcoma, temozolomide may be considered a therapeutic option as a single-agent.


Assuntos
Sarcoma , Neoplasias de Tecidos Moles , Humanos , Pessoa de Meia-Idade , Idoso , Temozolomida , Terapia de Salvação , Estudos Retrospectivos , Sarcoma/patologia
18.
Med J Malaysia ; 79(2): 196-202, 2024 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-38553926

RESUMO

OBJECTIVE: The standard treatment for regional failure in nasopharyngeal carcinoma (NPC) is the radical neck dissection (RND). Our study sought to determine if magnetic resonance imaging (MRI) may accurately predict nodal involvement to allow selected levels of neck dissection to be preserved. STUDY DESIGN AND SETTING: We analysed retrospectively all NPC patients in our centre undergoing neck dissections as salvage therapy for nodal recurrence. Nodal involvement based on the preoperative MRI was assessed and compared with postoperative histopathology. METHODS: This is a retrospective study conducted on patients in our centre with recurrent NPC from February 2002 to February 2017. Patients were identified from the database of the otolaryngology oncology division at our institution. Of these, 28 patients met all our inclusion and exclusion criteria. We calculated sensitivity and specificity as well as average number of nodes per patient. RESULTS: In our study, we calculated the false negative and false positive rates of preoperative MRI neck by levels. Overall sensitivity of MRI picking up disease by level was 76% and specificity was 86%. CONCLUSION: Based on our study, we will be missing a total of 10 (7.1%) diseased neck levels in eight (28.5%) patients. MRI alone, therefore, does not provide enough information to allow safe selective preservation of neck levels in surgical salvage of neck recurrences in NPC.


Assuntos
Neoplasias Nasofaríngeas , Esvaziamento Cervical , Humanos , Esvaziamento Cervical/métodos , Carcinoma Nasofaríngeo/cirurgia , Estudos Retrospectivos , Neoplasias Nasofaríngeas/diagnóstico por imagem , Neoplasias Nasofaríngeas/cirurgia , Neoplasias Nasofaríngeas/patologia , Terapia de Salvação , Recidiva Local de Neoplasia/cirurgia , Metástase Linfática
20.
Eur J Clin Invest ; 54(6): e14192, 2024 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-38445798

RESUMO

BACKGROUND: High-intensity focused ultrasound (HIFU) emerged as a novel approach for the treatment of localized prostate cancer (PCa). However, prospective studies on HIFU-related outcomes and predictors of treatment failure (TF) remain scarce. MATERIALS AND METHODS: We conducted a multinational prospective cohort study among patients undergoing HIFU therapy for localized, low- to intermediate-risk PCa. Follow-up data on serial prostate specific antigen (PSA), multi-parametric magnetic resonance imaging (mpMRI), targeted/systematic biopsies, adverse events and functional outcomes were collected. The primary endpoint was TF, defined as histologically confirmed PCa requiring whole-gland salvage treatment. Uni- and multi-variable adjusted hazard ratios (HRs) were calculated using Cox proportional hazard regression models. RESULTS: At baseline, mean (standard deviation) age was 64.14 (7.19) years, with the majority of patients showing T-stage 1 (73.9%) and International Society of Urological Pathology grading system Grade 2 (58.8%). PSA nadir (median, 1.70 ng/mL) was reached after 6 months. Of all patients recruited, 16% had clinically significant PCa, as confirmed by biopsy, of which 13.4% had TF. Notably, T-stage and number of positive cores at initial biopsy were independent predictors of TF during follow-up (HR [95% CI] 1.27 [1.02-1.59] and 5.02 [1.80-14.03], respectively). Adverse events were minimal (17% and 8% early and late adverse events, respectively), with stable or improved functional outcomes in the majority of patients. CONCLUSIONS: This interim analysis of a multinational study on HIFU therapy for the management of low-to-intermediate-risk PCa reveals good functional outcomes, minimal adverse events and low incidence of TF over the short-term. Data on long-term outcomes, specifically as it relates to oncological outcomes, are awaited eagerly.


Assuntos
Antígeno Prostático Específico , Neoplasias da Próstata , Humanos , Masculino , Neoplasias da Próstata/terapia , Neoplasias da Próstata/patologia , Pessoa de Meia-Idade , Idoso , Antígeno Prostático Específico/metabolismo , Antígeno Prostático Específico/sangue , Estudos Prospectivos , Ultrassom Focalizado Transretal de Alta Intensidade , Falha de Tratamento , Modelos de Riscos Proporcionais , Terapia de Salvação/métodos , Ablação por Ultrassom Focalizado de Alta Intensidade/métodos , Imageamento por Ressonância Magnética Multiparamétrica , Gradação de Tumores , Estudos de Coortes
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