- [A comparison of C+SCAV and SEAM conditioning regimens in efficacy and safety in autologous hematopoietic stem cell transplantation for non-Hodgkin's lymphoma patients]. [Journal Article]Zhonghua Xue Ye Xue Za Zhi. 2022 Aug 14; 43(8):668-673.ZX
- Objective: This study aimed to compare the efficacy and safety of cladribine, smustine, etoposide, cyclophosphamide, and cytarabine (C+SCAV) and smustine, etoposide, cytarabine, and melphalan (SEAM) conditioning regimens in autologous stem cell transplantation (auto-HSCT) for non-Hodgkin's lymphoma (NHL) . Methods: A retrospective analysis was conducted on 61 NHL patients who received auto-HSCT i…
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- Hydroxyl transfer versus cyclization reaction in the gas phase: Sequential loss of NH3 and CH2CO from protonated phenylalanine derivatives. [Journal Article]Front Chem. 2022; 10:1094329.FC
- Collisional activation of protonated phenylalanine derivatives deamination products leads to hydroxyl skeletal rearrangement versus cyclization reaction, and to form hydroxylbenzyl cation via elimination of CH2CO. To better clarify this unusual fragmentation reaction, accurate mass measurements experiments, native isotope experiments, multiple-stage mass spectrometry experiments, different substi…
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- Biochemical measures of ovarian function in female survivors of retinoblastoma treated with intra-arterial melphalan: an initial report. [Journal Article]Ophthalmic Genet. 2022 Dec; 43(6):806-808.OG
- Since 2006, ophthalmic artery chemosurgery (OAC) has been used for ocular-sparing treatment of retinoblastoma. Systemic exposure to melphalan is known to cause ovarian dysfunction, but the effect of melphalan-based OAC has not yet been determined. Here, we assess biochemical and symptomatic measures of ovarian function in a cohort of pubertal female survivors of retinoblastoma treated with melpha…
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- Daratumumab, pomalidomide, and dexamethasone (DPd) followed by high dose chemotherapy-Autologous Stem Cell Transplantation leads to superior outcomes when compared to DPd-alone for patients with Relapsed Refractory Multiple Myeloma. [Journal Article]Transplant Cell Ther. 2023 Jan 19 [Online ahead of print]TC
- CONCLUSIONS: Patients with RRMM who responded to DPd and underwent HDT-ASCT demonstrated superior depth and duration of remission compared to those who received DPd-alone. Although DPd followed by ASCT is associated with more cytopenias and gastrointestinal toxicities, this treatment appears to be overall safe for patients with RRMM.
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- Safety and efficacy of autologous stem cell transplantation in dialysis-dependent myeloma patients-The DIADEM study from the chronic malignancies working party of the EBMT. [Journal Article]Bone Marrow Transplant. 2023 Jan 21 [Online ahead of print]BM
- The role of high-dose chemotherapy followed by autologous stem cell transplantation (ASCT) in the treatment of myeloma (MM) patients with severe and/or dialysis-dependent renal impairment remains uncertain. We report on the outcomes of 110 patients (median age 57 years) who had become dialysis-dependent pre-ASCT and who underwent a first ASCT between 1997 and 2017. Sixty-three (57%) patients had …
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- Outcome of clinical experience of introducing a patient with secondary systemic AL amyloidosis associated with multiple myeloma. [Journal Article]Carcinogenesis. 2023 Jan 20 [Online ahead of print]C
- This research is relevant, as AL-amyloidosis refers to a systemic type of disease characterised by aggregation of an improperly folded light chain of immunoglobulin, mainly in the heart and kidneys, causing organ failure. This study describes the clinical experience of introducing a patient with cardiac amyloidosis associated with multiple myeloma. A clinical case of a patient diagnosed with amyl…
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- Liposome-Encapsulated Melphalan Exhibits Potent Antimyeloma Activity and Reduced Toxicity. [Journal Article]
- Multiple myeloma (MM), a plasma cell cancer in bone marrow, remains an incurable disease. Melphalan, an alkylating agent, is a conventional anticancer drug that is still widely used for MM treatment in clinics. However, melphalan-induced organ toxicity and side effects are common. In this study, we loaded melphalan into a liposomal capsule and constituted liposomal melphalan (liposomal MEL). Lipo…
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- Peritransplant glucocorticoid haploidentical stem cell transplantation is a promising strategy for AML patients with high leukemic burden: Comparison with transplants using other donor types. [Journal Article]Transplant Cell Ther. 2023 Jan 11 [Online ahead of print]TC
- Using a murine haploidentical BMT model, we recently showed that peritransplant administration of glucocorticoid (GC) redistributed donor T cells from the gastrointestinal tract to bone marrow, which resulted in a significant reduction of GVHD while promoting GVL effects. Furthermore, in a retrospective clinical study of patients with AML undergoing transplantation in non-remission, we also showe…
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- Comparative Efficacy and Safety of Beam and Team Conditioning Regimens for Autologous Stem Cell Transplantation in Lymphoma Patients. [Journal Article]Transplant Proc. 2023 Jan 11 [Online ahead of print]TP
- CONCLUSIONS: To the best of our knowledge, this is one of the few studies in the literature that compared the TEAM and BEAM as conditioning regimens for ASCT in lymphoma patients. The 2 regimens may provide similar overall survival outcomes and have a comparable safety profile. Although the BEAM regimen may be associated with longer progression-free survival times, the difference may be covered by the similar survival after ASCT.
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- Autologous stem cell transplantation in multiple myeloma patients with renal impairment. [Journal Article]Ann Hematol. 2023 Jan 12 [Online ahead of print]AH
- Renal impairment (RI) used to exclude multiple myeloma (MM) patients from autologous stem cell transplantation (ASCT) for safety concerns. Here, we retrospectively reviewed 34 consecutively transplanted patients with creatinine clearance < 60 ml/min at ASCT in recent 5 years at our institution. Busulfan/cyclophosphamide and high-dose melphalan were both employed as conditioning regimens. We found…
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- High-dose chemotherapy followed by autologous stem cell transplantation for adult patients With first relapse of Ewing's sarcoma: A single institution experience. [Journal Article]
- The prognosis of patients with Ewing's sarcoma family of tumors (ESFT) relapse is poor; the 5-year overall survival (OS) is 13%. We evaluated the effectivity of high-dose therapy (HDT) with autologous stem cell transplantation (ASCT) in adult patients with ESFT relapse. Between January 2010 and January 2021, we retrospectively analyzed 20 patients with ESFT who received HDT upon relapse. A combin…
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- Tracking the Evolution of Therapy-Related Myeloid Neoplasms Using Chemotherapy Signatures. [Journal Article]Blood. 2023 Jan 10 [Online ahead of print]Blood
- Patients treated with cytotoxic therapies, including autologous stem cell transplantation, are at risk for developing therapy-related myeloid neoplasms (tMN). Pre-leukemic clones (i.e., clonal hematopoiesis; CH) are detectable years before the development of these aggressive malignancies, though the genomic events leading to transformation and expansion are not well-defined. Here, leveraging dist…
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- Combining Melphalan Percutaneous Hepatic Perfusion with Ipilimumab Plus Nivolumab in Advanced Uveal Melanoma: First Safety and Efficacy Data from the Phase Ib Part of the Chopin Trial. [Journal Article]Cardiovasc Intervent Radiol. 2023 Jan 09 [Online ahead of print]CI
- CONCLUSIONS: Combining M-PHP with IPI/NIVO was safe in this small cohort of patients with mUM at a dose of IPI 1 mg/kg and NIVO 3 mg/kg.
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- High-Dose Melphalan Treatment Significantly Increases Mutational Burden at Relapse in Multiple Myeloma. [Journal Article]Blood. 2023 Jan 05 [Online ahead of print]Blood
- High Dose Melphalan (HDM) improves progression free survival (PFS) in multiple myeloma (MM), yet melphalan is a DNA damaging alkylating agent, so we assessed its mutational effect on surviving myeloma cells by analyzing paired MM samples collected at diagnosis and relapse from the IFM 2009 study. We performed deep whole genome sequencing on 68 patients, 43 treated with RVD (lenalidomide, bortezom…
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- Cross-neutralization and viral fitness of SARS-CoV-2 Omicron sublineages. [Journal Article]
- The rapid evolution of SARS-CoV-2 Omicron sublineages mandates a better understanding of viral replication and cross-neutralization among these sublineages. Here we used K18-hACE2 mice and primary human airway cultures to examine the viral fitness and antigenic relationship among Omicron sublineages. In both K18-hACE2 mice and human airway cultures, Omicron sublineages exhibited a replication ord…
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- Outcomes After Salvage Autologous Hematopoietic Cell Transplant for Patients With Relapsed/Refractory Multiple Myeloma: A Single-Institution Experience. [Journal Article]Clin Lymphoma Myeloma Leuk. 2022 Dec 05 [Online ahead of print]CL
- CONCLUSIONS: For MM patients deriving durable remission after their AHCT1, sAHCT2 was safe and resulted in deep and durable remissions.
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- Evaluating the Impacts of CYP3A4*1B and CYP3A5*3 Variations on Pharmacokinetic Behavior and Clinical Outcomes in Multiple Myeloma Patients With Autologous Stem Cell Transplant. [Journal Article]
- CONCLUSIONS: CYP3A4*1B/*1B and CYP3A5*3/*3 variations might influence melphalan therapy in MM patients through yet-to-be-identified mechanisms.
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- Impact of Conditioning Regimen and Graft-versus-Host Disease Prophylaxis on The Outcome of Haploidentical Peripheral Blood Stem Cell Transplantation for High-Risk Severe Aplastic Anemia in Children and Young Adults: A Report from the Pediatric... [Journal Article]Transplant Cell Ther. 2022 Dec 23 [Online ahead of print]TC
- Allogenic hematopoietic cell transplantation (HCT) is the best curative approach for patients with severe aplastic anemia (SAA). The outcomes of HCT from haploidentical family donors (HFDs) have improved, making it a feasible option for patients lacking an HLA-identical donor. However, data on HFD-HCT for younger patients with SAA is sparse. In this multicenter retrospective study, we evaluated t…
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- The BLM helicase is a new therapeutic target in multiple myeloma involved in replication stress survival and drug resistance. [Journal Article]
- Multiple myeloma (MM) is a hematologic cancer characterized by accumulation of malignant plasma cells in the bone marrow. To date, no definitive cure exists for MM and resistance to current treatments is one of the major challenges of this disease. The DNA helicase BLM, whose depletion or mutation causes the cancer-prone Bloom's syndrome (BS), is a central factor of DNA damage repair by homologou…
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- The Combination of Panobinostat and Melphalan for the Treatment of Patients with Multiple Myeloma. [Journal Article]
- Histone deacetylase inhibitors show synergy with several genotoxic drugs. Herein, we investigated the biological impact of the combined treatment of panobinostat and melphalan in multiple myeloma (MM). DNA damage response (DDR) parameters and the expression of DDR-associated genes were analyzed in bone marrow plasma cells (BMPCs) and peripheral blood mononuclear cells (PBMCs) from 26 newly diagno…
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- Clinical Impact of Melphalan Pharmacokinetics on Transplantation Outcomes in Children Undergoing Hematopoietic Stem Cell Transplantation. [Journal Article]
- Melphalan is widely used for hematopoietic stem cell transplantation (HSCT) conditioning. However, the relationship between its pharmacokinetic (PK) and transplantation outcomes in children has not been thoroughly investigated. We prospectively analyzed the relationship between melphalan area under the curve (AUC) and transplantation outcome and examined the development of a predictive model for …
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- Upfront autologous haematopoietic stem-cell transplantation versus carfilzomib-cyclophosphamide-dexamethasone consolidation with carfilzomib maintenance in patients with newly diagnosed multiple myeloma in England and Wales (CARDAMON): a randomised, phase 2, non-inferiority trial. [Journal Article]Lancet Haematol. 2022 Dec 15 [Online ahead of print]LH
- CONCLUSIONS: KCd did not meet the criteria for non-inferiority compared with autologous HSCT, but the marginal difference in progression-free survival suggests that further studies are warranted to explore deferred autologous HSCT in some subgroups, such as individuals who are MRD negative after induction.
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- Carfilzomib induction, consolidation, and maintenance with or without autologous stem-cell transplantation in patients with newly diagnosed multiple myeloma: pre-planned cytogenetic subgroup analysis of the randomised, phase 2 FORTE trial. [Randomized Controlled Trial]Lancet Oncol. 2023 Jan; 24(1):64-76.LO
- CONCLUSIONS: This preplanned analysis of the FORTE trial showed that carfilzomib-based induction-intensification-consolidation regimens are effective strategies in patients with standard risk (zero HRCA) and high-risk (one HRCA) myeloma, resulting in similar rates of progression-free survival and 1-year sustained minimal residual disease negativity. Despite promising progression-free survival, patients with ultra-high-risk disease (those with 2 or more HRCA) still have an increased risk of progression and death and therefore represent an unmet medical need.
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- Comparison of two chemotherapy regimens in patients with newly diagnosed Ewing sarcoma (EE2012): an open-label, randomised, phase 3 trial. [Randomized Controlled Trial]
- Internationally, a single standard chemotherapy treatment for Ewing sarcoma is not defined. Because different chemotherapy regimens were standard in Europe and the USA for newly diagnosed Ewing sarcoma, and in the absence of novel agents to investigate, we aimed to compare these two strategies.
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- Reduced-intensity conditioning is effective for allogeneic hematopoietic stem cell transplantation in infants with MECOM-associated syndrome. [Journal Article]Int J Hematol. 2022 Dec 14 [Online ahead of print]IJ
- Mutations in the MECOM encoding EVI1 are observed in infants who have radioulnar synostosis with amegakaryocytic thrombocytopenia. MECOM-associated syndrome was proposed based on clinical heterogeneity. Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for progressive bone marrow failure. However, data regarding allogeneic HSCT for this rare disease are limited. We…
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- Effects of the SLCO1B1 A388G single nucleotide polymorphism on the development, clinical parameters, treatment, and survival of multiple myeloma cases in a Polish population. [Journal Article]Mol Biol Rep. 2022 Dec 07 [Online ahead of print]MB
- CONCLUSIONS: The A388G polymorphism does not seem to affect the increased risk of the development of multiple myeloma. However, the occurrence of the GG genotype may prolong of patients overall survival in the case of Melphalan-Prednisone therapy.
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- [Anesthesiological and postinterventional management in percutaneous hepatic melphalan perfusion (chemosaturation)]. [Review]Anaesthesiologie. 2022 Dec 07 [Online ahead of print]A
- Percutaneous hepatic melphalan perfusion (PHMP) is a last-line treatment of inoperable primary or secondary liver tumors. Selective perfusion and saturation (chemosaturation) of the liver with the chemotherapeutic agent melphalan is performed via catheterization of the hepatic artery without affecting the rest of the body with its cytotoxic properties. Using an extracorporeal circulation and ball…
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- Quantitative evaluation of therapy options for relapsed/refractory diffuse large B-cell lymphoma: A model-based meta-analysis. [Review]Pharmacol Res. 2023 Jan; 187:106592.PR
- New therapies for relapsed/refractory diffuse large B-cell lymphoma (r/rDLBCL) have emerged in recent years, but there have been no comprehensive quantitative comparisons of the efficacy of these therapies. In this study, the efficacy characteristics of 11 types of treatment strategy and 63 treatment regimens were compared by model based meta-analysis. We found that compared with monotherapy, ass…
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- Single Center Experience of Autologous Stem Cell Transplantation in Patients with Systemic Light Chain Amyloidosis in Korea. [Journal Article]Clin Lymphoma Myeloma Leuk. 2022 Oct 22 [Online ahead of print]CL
- CONCLUSIONS: ASCT is an effective treatment option for eligible patients with AL amyloidosis. Achieving hematologic CR is essential for long-term survival.
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