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Histopathologic findings after selective ophthalmic arterial injection of melphalan for retinoblastoma.

PURPOSE: The aim is to describe histopathologic observations in eyes enucleated after selective ophthalmic arterial injection (SOAI) of melphalan for retinoblastoma (RB). STUDY DESIGN: This is retrospective clinical study. PATIENTS AND METHODS: Histopathologic analysis of 14 eyes (13 patients) from May 2008 through January 2015 at Chang Gung Memorial Hospital. RESULTS: The eyes after SOAI were enucleated due to tumor viability (n = 7, 2 with vitreous hemorrhage), neovascular glaucoma (n = 4), lens drop to vitreous with total hyphema and elevated intraocular pressure (n = 1), retinal detachm…

Intravitreal melphalan for persistent or recurrent retinoblastoma vitreous seeds: preliminary results.

IMPORTANCE: Recurrent or persistent vitreous seeds following treatment of retinoblastoma poses difficult management and often leads to enucleation. OBJECTIVE: To describe the technique and evaluate the efficacy and complications of intravitreal melphalan for vitreous seeding from retinoblastoma. DESIGN, SETTING, AND PARTICIPANTS: This retrospective noncomparative analysis was conducted at a tertiary referral center. The study included 11 consecutive eyes of 11 patients with viable persistent or recurrent vitreous seeds following treatment of retinoblastoma. INTERVENTIONS: All eyes received …

Pharmacokinetics & drug resistance of melphalan in regional chemotherapy: ILP versus ILI.

Two forms of regional chemotherapy for the treatment of advanced melanoma or sarcoma of the extremity are isolated limb perfusion (ILP) and the more recently described isolated limb infusion (ILI). Melphalan is the most commonly employed agent in both ILP and ILI, although it is often used in conjunction with other cytotoxic and/or biologic therapies. While ILP and ILI are far more effective for the treatment of extremity disease than is systemic therapy, there is still significant room for improvement in outcomes, from the standpoint of both response rate and toxicity. An understanding of the…

Scleromyxedema: role of high-dose melphalan with autologous stem cell transplantation.

Scleromyxedema, the most severe manifestation of the spectrum of lichen myxedematosus, is characterized by cutaneous mucinosis, extracutaneous manifestations, and a monoclonal gammopathy. Seven of 8 patients evaluated at our center were treated with high-dose melphalan (180 mg/m(2) intravenously) and autologous peripheral blood stem cell transplantation, with marked improvement of gastrointestinal, central nervous system, pulmonary manifestations, and Karnofsky performance status. Five patients obtained a cutaneous complete remission and 2 patients had partial remissions. Three patients with s…

Vitamin E Oil Incorporated Liposomal Melphalan and Simvastatin: Approach to Obtain Improved Physicochemical Characteristics of Hydrolysable Melphalan and Anticancer Activity in Combination with Simvastatin Against Multiple Myeloma.

The objective of this research was to develop vitamin E oil (VEO)-loaded liposomes for intravenous delivery and to study the VEO effect on melphalan (MLN) loading, release, and stability. Further, the research aim was to determine the in vitro anticancer activity and in vivo systemic toxicity of MLN and simvastatin (SVN) combinations, for repurposing SVN in multiple myeloma. The liposomes were prepared by thin-film hydration technique. The optimized liposomes were surface modified with Pluronic F108, lyophilized, and evaluated for mean particle size, MLN content and release behavior, and in vi…

Анализ экономической эффективности даратумумаба плюс бортезомиба, мелфалана и преднизона против бортезомиба, мелфалана и преднизона у пациентов с недавно поставленным диагнозом множественной миеломы, не имеющей права на трансплантацию стволовых клеток.

Цель этого исследования заключалась в изучении экономической эффективности бортезомиба, мелфалана и преднизона с использованием даратумумаба (D-VMP) против борцезомиба, мелфалана и преднизона (VMP) у пациентов с недавно поставленным диагнозом множественной миеломы (NDMM), которые были лишены права на автоматическую трансплантацию стволовых клеток (ASCT). Была разработана модель MetodsA Маркова и three Включены только состояние здоровья, отсутствие прогресса в выживании (ПФС), прогрессивные заболевания (ПЗ) и смерть. Продолжительность цикла была установлена следующим образом: 1.5 месяцев и врем

Совпадение в аннотации оригинала
ObjectivesThe aim of this study was to investigate the cost-effectiveness of bortezomib, melphalan and prednisone with daratumumab (D-VMP) versus bortezomib, melphalan and prednisone (VMP) in patients with newly diagnosed multiple myeloma (NDMM) who were ineligible for autologous stem-cell transplantation (ASCT).MethodsA Markov model was established and thre…

A pilot study of intraoperative melphalan to prevent recurrent PVR: the IOMPVR study.

BACKGROUND: Proliferative vitreoretinopathy (PVR) is a major cause of failure in cases of retinal detachment (RD) repair. Intravitreal melphalan, a known inhibitor of cellular proliferation, offers a novel therapeutic approach to reduce PVR recurrence and improve outcomes. We evaluated the safety and efficacy of 5 µg/0.1 ml intravitreal melphalan at the end of pars plana vitrectomy (PPV) before silicone oil (SO) injection in eyes with primary PVR related to rhegmatogenous retinal detachments (RRDs) with a minimal 90-day follow-up period. METHODS: This prospective, cross-sectional, interventio…

Alemtuzumab, Fludarabine, Melphalan, and Thiotepa Conditioning for Transplantation in Inborn Errors of Immunity.

BACKGROUND: Inborn errors of immunity (IEI) are characterized by impaired lymphoid or myeloid immune cell function, resulting in recurrent i … rough -4 (150 mg/m² or 5 mg/kg in patients weighing <10 kg), thiotepa on day -4 (8 mg/kg total) and melphalan on day -3 (140 mg/m² or 4.7 mg/kg in patients weighing <10 kg). Graft sources included bone marrow (BM), peripheral blood stem cells (PBSCs), or umbilical cord blood (UCB). RESULTS: Twenty-eight pediatric and young adult patients with IEI received …

Efficacy of a prolonged stability melphalan formulation for intra-arterial treatment of retinoblastoma.

BACKGROUND: Melphalan, which is poorly soluble at room temperature, is widely used for the treatment of retinoblastoma by selective ophthalmic artery infusion. Evomela, a propylene glycol-free formulation of melphalan with improved solubility and stability, has recently been used as an alternative.To compare the safety and efficacy of Evomela with standard-formulation melphalan (SFM) in the treatment of retinoblastoma by selective ophthalmic artery infusion. METHODS: We performed a retrospective case-control study of patients with retinoblastoma undergoing selective ophthalmic artery infusion…

Rituximab combined with a small dose of melphalan for a refractory follicular lymphoma patient.

A 48-year-old male patient with follicular lymphoma, grade II, stage IV, was treated with CHOP, ESHAP and MACOP-B, resulting in partial remission. After 9 months, the disease progressed and several chemotherapy agents, including three courses of rituximab combined with etoposide, sobuzoxane or methotrexate, only resulted in a stable disease response. However, the fourth course of rituximab combined with a small dose of melphalan produced excellent results and the complete response continued for more than 15 months. It is possible that these two drugs may act synergistically.

Deletion of 5q31 and 7q31 in patients with stable melphalan treated multiple myeloma.

Multiple myeloma represents a malignant proliferation of plasma cells derived from a single clone. It is well known that alkylating agents are capable of inducing myelodysplastic syndromes (MDS) and acute myelocytic leukemias (AML). This risk of both diseases in patients with multiple myeloma has been estimated to be 10-20% after 10 years. We aimed to evaluate the time course and the type of genetic abnormalities in melphalan-treated patients in the chronic stage of the disease. We applied fluorescence in situ hybridization methods with probes to 5q31 and 7q31 to mononuclear peripheral blood l…

Targeting MCM6 Enhances Melphalan Chemosensitivity in Retinoblastoma by Modulating DNA Damage Response.

PURPOSE: This study aimed to investigate the role of minichromosome maintenance complex component 6 (MCM6), a DNA replication licensing factor, in retinoblastoma progression and its impact on melphalan chemosensitivity. METHODS: MCM6 expression patterns were analyzed using single-cell RNA sequencing (scRNA-seq) of retinoblastoma and validated in patient tumors, including specimens obtained after failed melphalan therapy. Stable MCM6 knockdown cell lines were established for proliferation and cell-cycle assays, DNA damage analyses, and chemosensitivity testing. In vivo xenograft models were em…

Indisulam synergizes with melphalan to inhibit Multiple Myeloma malignancy via targeting TOP2A.

Multiple myeloma (MM) is the second most prevalent hematologic malignancy which remains uncurable. Numerous drugs have been discovered to inhibit MM cells. Indisulam, an aryl sulfonamide, has a potent anti-myeloma activity in vitro and in vivo. This study aims to explore the new mechanism of indisulam and investigate its potential use in combination with melphalan. We examined DNA damage in MM cells through various methods such as western blotting (WB), immunofluorescence, and comet assay. We also identified the role of topoisomerase IIα (TOP2A) using bioinformatic analyses. The impact of indi…

Synthesis, 99m Tc-labeling, and preliminary biological evaluation of DTPA-melphalan conjugates.

Melphalan (MFL) is a typical nitrogen mustard for the treatment of many types of cancer. For the purpose to develop novel 99m Tc-labeled tumor imaging agents with SPECT, MFL was directly labeled by 99m Tc using diethylene triamine pentacetate acid (DTPA) as bifunctional chelating agent. The novel ligands were successfully synthesized by conjugation of DTPA to MFL to get monosubstituted DTPA-MFL and bis-substituted DTPA-2MFL. Radiolabeling was performed in high yield to get 99m Tc-DTPA-MFL and 99m Tc-DTPA-2MFL, respectively, which were hydrophilic and stable at room temperature. The high initia…

Iron Overload Exacerbates Busulfan-Melphalan Toxicity Through a Pharmacodynamic Interaction in Mice.

PURPOSE: Busulfan-melphalan high-dose chemotherapy followed by autologous stem cell transplantation is an essential consolidation treatment of high-risk neuroblastoma in children. Main treatment limitation is hepatic veno-occlusive disease, the most severe and frequent extra-hematological toxicity. This life threatening toxicity has been related to a drug interaction between busulfan and melphalan which might be increased by prior disturbance of iron homeostasis, i.e. an increased plasma ferritin level. METHODS: We performed an experimental study of busulfan and melphalan pharmacodynamic and …

Recommend upfront consolidation with high-dose melphalan and autologous stem cell support.

The overall goal of treatment for multiple myeloma (MM) is to prolong survival for as long as possible with the minimal treatment burden for each patient. Consolidation with upfront high-dose melphalan and autologous hematopoietic stem cell transplantation (ASCT) can prolong progression-free and overall survival by deepening response. It thereby can improve quality of life by allowing patients to have a time on maintenance therapy along with infrequent office visits, making it cost-effective in many situations. The impact of minimal residual disease (MRD) negativity is important, but further s…

Isolated hepatic perfusion with 200 mg melphalan for advanced noncolorectal liver metastases.

PURPOSE: The liver is one of the most common sites for metastatic solid tumors. If the liver is the only site of metastatic disease, regiona … , 1 hepatocellular carcinoma, and 1 high-grade sarcoma) were treated with a 60-min IHP using 200 mg melphalan. Patients were monitored for toxicity, response according to response evaluation criteria in solid tumors (RECIST) criteria, and survival. RESULTS: One melanoma patient was not perfused due to insufficient isolation of the liver. There was no treat …

Successful heart transplantation following melphalan plus dexamethasone therapy in systemic AL amyloidosis.

Recurrence in the allograft and progression in other organs increase mortality after cardiac transplantation in AL amyloidosis. Survival may be improved after suppression of monoclonal light chain (LC) production following high dose melphalan and autologous stem cell transplantation (HDM/ASCT). However, because of high treatment related mortality, this tandem approach is restricted to few patients without significant extra-cardiac involvement. A diagnosis of systemic AL amyloidosis was established in a 45-year old patient with congestive heart failure related to restrictive cardiomyopathy, nep…

Safety and Efficacy of Intravitreal Chemotherapy (Melphalan) to Treat Vitreous Seeds in Retinoblastoma.

Background: Active vitreous seeds in eyes with retinoblastoma (Rb) adversely affects the treatment outcome. This study aimed to investigate the safety and efficacy of intravitreal melphalan chemotherapy (IViC) as a treatment for recurrent and refractory vitreous seeds in patients with Rb. Methods: We used a retrospective non-comparative study of patients with intraocular Rb who had vitreous seeds and were treated by IViC (20-30 μg of melphalan) using the safety-enhanced anti-reflux technique. Tumor response, ocular toxicity, demographics, clinical features, and survival were analyzed. Results:…

Melphalan and prednisone plus thalidomide or placebo in elderly patients with multiple myeloma.

In this double-blind, placebo-controlled study, 363 patients with untreated multiple myeloma were randomized to receive either melphalan-prednisone and thalidomide (MPT) or melphalan-prednisone and placebo (MP). The dose of melphalan was 0.25 mg/kg and prednisone was 100 mg given daily for 4 days every 6 weeks until plateau phase. The dose of thalidomide/placebo was escalated to 400 mg daily until plateau phase and thereafter reduced to 200 mg daily until progression. A total of 357 patients were analyzed. Partial response was 34% and 33%, and very good partial response or better was 23% and 7…