thalidomide / Generic mfg. - LARVOL DELTA

Intestinal Behçet's disease presenting with intestinal obstruction misdiagnosed as Crohn's disease: a case report. (PubMed, J Med Case Rep) - "Given the strong resemblance between intestinal Behçet's disease and Crohn's disease, especially in the absence of typical extraintestinal manifestation, differentiation requires comprehensive endoscopic evaluation and systematic histopathological examination. Although the specific treatment regimen remains controversial, early diagnosis and timely initiation of medical therapy are crucial for improving prognosis."

Journal • Crohn's disease • Gastroenterology • Gastrointestinal Disorder • Immunology • Inflammatory Bowel Disease • Rare Diseases

Long-term HIV remission of a perinatally infected individual, following a hematopoietic cell transplantation from a CCR5Δ32 homozygous donor for multiple myeloma: The kyiv patient (ASH 2025) - "Complete remission (CR) was achieved after one cycle of melphalan/prednisone and local radiotherapy, but an abdominal relapse occurred six months later. Despite subsequent lines of therapy (bortezomib(bort)/lenalidomide(lena)/dexamethasone(dexa) and bendamustine/bort/dexa), the extramedullary multiple myeloma (MM) progressed. Remission was achieved after dexa/thalidomide/cisplatin/doxorubicin/cyclophosphamide/etoposide (DT-PACE) and an autologous HCT performed on 1/2021...Allogeneic HCT was performed in 8/2022 after fludarabine/melphalan conditioning and anti-thymocyte globulin from a CCR5-Δ32homozygous, unrelated HLA-matched donor; cyclosporine/methotrexate was utilized as graft-versus-host diseas e (GVHD) prophylaxis... To our knowledge, this represents the first report of ART-free HIV RNA suppression following allogeneic HCT with a CCR5Δ32homozygous donor in an individual perinatally infected with HIV. Despite the differences in the latent reservoir and the mechanism..."

Clinical • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Human Immunodeficiency Virus • Immunology • Infectious Disease • Multiple Myeloma • Plasmacytoma • Transplantation • CCR5 • CD4 • CD8

An evolving landscape – how changing conditioning regimes can improve myelofibrosis transplant outcomes. (ASH 2025) - "Of the 16 cases, seven received Fludarabine/Busulphan/Antithymocyte globulin (Flu/Bu/ATG) conditioning (Fludarabine 30mg/m 2 day -9 to day -5, Busulphan 3.2mg/kg on day -4 and day -3 and rabbit ATG 2.5mg/kg on day -2 and day -1), seven underwent Fludarabine/Cyclophosphamide (Flu/Cy) conditioning (Fludarabine 25mg/m 2 day -6 to day -2 and Cyclophosphamide 1g/m 2 on day -3 and day -2), and two received Fludarabine/Melphalan (Flu/Mel) (Fludarabine 25mg/m 2 day -6 to day -2 and Melphalan 100mg/m 2 on day -2)...Therapies included Ruxolitnib, Momelotinib, splenic irradiation, Hydroxycarbamide, Danazol, Interferon, Cyclophosphamide, Anagrelide, Thalidomide, Panobinostat and Navitoclax...Although limited by a small sample size, these findings indicate that the Flu/Bu/ATG conditioning regimen may be associated with improved outcomes, thereby supporting its adoption as the preferred approach for patients undergoing allogeneic stem cell transplantation for myelofibrosis as per BSH..."

Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Infectious Disease • Myelofibrosis • Pneumonia • Respiratory Diseases • Transplantation • ASXL1 • CALR • JAK2 • SF3B1 • U2AF1

First results of aponermin(Apo) improving teclistamab (tec)/talquetamab (tal) in patients (pts) with Relapsed/Refractory multiple myeloma (RRMM) (ASH 2025) - P=N/A | " As of August 3, 2025, 6 pts received Apo+ Tec (including 1pts with Tal causing resistant of BCMA) +Thalidomide. In this first combination study of Apo+BCMA-/GPRC5D-targeted bispecific antibody, at the RP2R has a manageable safety profile consistent with each of the monotherapies. In dose level 2, a 100% ORR was observed in pts with advanced RRMM at the RP2R, and an ORR of 66.7% was achieved in pts with EMD, a high-risk population with unmet need, supporting further evaluation of the combination. Clinical trial information: ChiCTR2500106279."

Clinical • Hematological Disorders • Hematological Malignancies • Infectious Disease • Inflammation • Multiple Myeloma • Nephrology • Neutropenia • Renal Disease • Thrombocytopenia

Early intervention in smoldering multiple myeloma: A systematic review of comparative prospective trials (ASH 2025) - "Two studies were phase I/II trials, one comparing the use of dose-escalated multipeptide PVX-410 vaccine +/- lenalidomide (R) in SMM patients, and the other comparing dose-escalated anakinra +/-Lenalidomide/dexamethasone (Rd)...Two studies were phase II RCTs; one compared Rd +/- Carfilzomib (K) in 58 high-risk SMM patients, with statistically significantly higher 3-year PFS in KRd of 94% versus Rd of 40%; p<0.001, across a median follow-up period of 34 months...The second phase II RCT included 20 intermediate- and high-risk SMM patients treated with Iberdomide (I) +/- d, with ORR of 84.6% versus 80% in the Id versus I group...One phase III RCT compared zoledronic acid +/- thalidomide in a total of 68 SMM patients, with a median TTP of 2.4 versus 1.2 years, 1-year PFS of 85% versus 55%, and 1-year ORR of 37% versus no confirmed response during the median follow-up of 5.9 years... Preliminary evidence from comparative trials suggests that early treatment of..."

Clinical • Review • Hematological Malignancies • Multiple Myeloma • Smoldering Multiple Myeloma

Efficacy and safety of aponermin combined with immunomodulators in relapsed/refractory multiple myeloma (ASH 2025) - "Treatment regimens included aponermin plus carfilzomib, thalidomide, and dexamethasone (Apo-KTd); aponermin plus carfilzomib, pomalidomide, and dexamethasone (Apo-KPd); aponermin plus pomalidomide and dexamethasone (Apo-Pd); aponermin plus thalidomide and dexamethasone (Apo-Td), and other combination therapies based on aponermin. Conclusion Aponermin combined with immunomodulators demonstrates good tolerability and definite efficacy for RRMM, especially for long-term treatment. Patients with high-risk cytogenetics, extramedullary lesions, and those who have failed multiple lines of treatment can still benefit."

Clinical • Immunomodulating • IO biomarker • Hematological Malignancies • Multiple Myeloma

Outcomes of venous thromboembolism in multiple myeloma: A nationwide analysis of anticoagulation strategies (ASH 2025) - "The current guideline from ASH has a conditional recommendation regarding the use of anticoagulation for VTE prophylaxis in ambulatory patients with MM receiving lenalidomide-, thalidomide-, or pomalidomide-based regimens. Conclusion MM patients who developed VTE without any prophylactic antithrombotic therapy experienced worse outcomes, including longer hospitalizations, higher healthcare costs, and increased mortality. Use of aspirin or anticoagulants was associated with improved clinical outcomes, underscoring the importance of reinforcing thromboprophylaxis in MM patients."

Cardiovascular • Hematological Malignancies • Multiple Myeloma • Venous Thromboembolism

Requirements for achievement of treatment-free remission in long-term survivor of multiple myeloma (ASH 2025) - "The first patient maintained treatment-fre e remission for 14 years after discontinuing thalidomide (Thal) maintenance, following remission induction and tandem autologous stem cell transplantation (ASCT)...This clinical status represents the closest approximation to a cure in MM. Given the rarity of such cases, we need further analysis of this patient subgroup."

Clinical • IO biomarker • Hematological Malignancies • Multiple Myeloma • ASXL1 • HLA-B • KRAS • TET2 • TP53

Safety and efficacy of carfilzomib-based combinations in relapsed/refractory multiple myeloma patients in a real-world Setting : Results of a french single academic center retrospective study. (ASH 2025) - "All patients had been exposed to IMiDs (lenalidomide, pomalidomide, thalidomide), 89% to proteasome inhibitors (bortezomib, carfilzomib, ixazomib), and 64% to anti-CD38 monoclonal antibodies (daratumumab, isatuximab). Initial weekly dosing of K followed by spacing out infusions in responding patients led to better tolerance. Currently, K-based combinations also represent interesting options as bridging therapy to CAR T-cells therapies."

Real-world • Real-world evidence • Retrospective data • Cardiovascular • Congestive Heart Failure • Coronary Artery Disease • Gastrointestinal Disorder • Heart Failure • Hematological Malignancies • Hypertension • Multiple Myeloma

Outcomes of infusional chemotherapy regimens in multiple myeloma: A retrospective review at a single center from 2018 to 2025 (ASH 2025) - "Despite emergence of novel agents, the use of traditional multi-agent infusional regimens such as DCEP (dexamethasone, cyclophosphamide, etoposide, cisplatin) and V(T)D-PACE (bortezomib, thalidomide, dexamethasone, cisplatin, doxorubicin, cyclophosphamide, etoposide) remain critical in select clinical scenarios. These regimens continue to be valuable in providing rapid cytoreduction or as a bridge to definitive therapy. Larger sample sizes are required in order to perform statistical analysis on subgroups to better characterize differences in therapy with respect to progression free survival, overall response rate, and time to response."

Retrospective data • Review • Hematological Malignancies • Leukemia • Multiple Myeloma • Plasma Cell Leukemia

Successful melphalan flufenamide (melflufen) therapy in patients with severe renal insufficiency (ASH 2025) - "Before melflufen, the patient received 5 prior therapies all of which were discontinued due to different reasons: VMP (10 mo) – progredient disease (PD), Rd (3 mo) – drug exanthema, Vd (7 mo) - PD, DaraKd (10 mo) – heart insufficiency, and DaraPd (4 mo) which was adjusted to daratumumab monotherapy due to side effects on pomalidomide...In total, 9 different prior therapies were administered and discontinued before the use of melflufen: thalidomide/doxorubicine/dexamethasone (12 mo) with thalidomide as a maintenance therapy (2 y) – PD, Vd (12 mo) – polyneuropathy, bendamustine/prednisolone (5 mo) - PD, KRd (4 mo) – VGPR, discontinuation due to patient's request, EloRd (9 mo) – VGPR, DaraRd (2 y) and daratumumab monotherapy (4 mo) - PD, IsaPomd (5 mo) – PD, and liposomal doxorubicine/bortezomib (2 mo)... Both clinical cases showed that melphalan flufenamide (melflufen) can be administered in a reduced dose of e.g. 20 mg and is a highly effective and well-tolerated..."

Clinical • Cardiovascular • Congestive Heart Failure • Diabetes • Heart Failure • Hematological Malignancies • Infectious Disease • Metabolic Disorders • Multiple Myeloma • Musculoskeletal Diseases • Nephrology • Orthopedics • Pulmonary Arterial Hypertension • Renal Disease • Type 2 Diabetes Mellitus • B2M

Survival and clinical response to bortezomib, thalidomide, and dexamethasone in patients with multiple myeloma at a peruvian tertiary care center: A real-world retrospective observational study (ASH 2025) - "Conclusions VTD-based regimens, regardless of dosing schedule, remain an effective first-line treatment option for multiple myeloma, particularly in healthcare systems with limited resources where access to high-cost therapies remains restricted. Furthermore, ASCT continues to be a standard consolidation strategy for eligible patients, enabling deeper responses and improving both OS and PFS."

Observational data • Real-world • Real-world evidence • Retrospective data • Cardiovascular • Chronic Kidney Disease • Diabetes • Diabetic Nephropathy • Endocrine Disorders • Hematological Malignancies • Hypertension • Metabolic Disorders • Multiple Myeloma • Nephrology • Renal Disease

Risk factors for varicella zoster virus reactivation in newly diagnosed multiple myeloma patients undergoing autologous stem cell transplantation (ASH 2025) - "Maintenance therapy consisted of lenalidomide in 53 (56%) patients, bortezomib for two years in 14 (15%) patients, thalidomide in 4 (4%) patients and other combination for the other 29 patients...Regarding VZV reactivation severity, 85% were grade 2, treated with oral aciclovir or valaciclovir, 2 (10%) grade 3 and one patient (5%) grade 4...This study is the first to describe, in a real-world cohort of MM patients undergoing ASCT, the prevalence and potential risk factors for VZV reactivation also in vaccinated patients. The possible role of prognostic biomarkers, namely B2M levels, in predicting VZV reactivation needs to be confirmed in larger datasets."

Clinical • IO biomarker • CNS Disorders • Hematological Disorders • Hematological Malignancies • Herpes Zoster • Leukemia • Multiple Myeloma • Plasma Cell Leukemia • Renal Disease • Transplantation • Varicella Zoster • B2M • CD4

Bone marrow adipose tissue is enriched in multiple myeloma cells (ASH 2025) - "In vitro , a 4-hour treatment of isolated fat-associated and aspirate-associated cells with bortezomib, melphalan, dexamethasone, or thalidomide revealed a high inter-patient heterogeneity, as in a subset of patients adipose-associated MM cells reacted less to treatment compared to MM cells from the aspirate (measured as percentage of viable MM cells after treatment). The specific molecular interactions between adipocytes, MM cells, and immune cells remain to be identified. Furthermore, as the amount (and presence) of BMAT is highly heterogenous per patient, the exact significance of fat-associated MM cells and their contribution to disease burden has to be assessed."

Hematological Malignancies • Monoclonal Gammopathy • Multiple Myeloma • FABP5 • IRF4 • NCAM1

Efficacy and safety of Golidocitinib-based first line regimen in peripheral T-cell lymphoma: A multicenter retrospective study (ASH 2025) - "All patients received golidocitinib combined with chemotherapy, including CHOP-like regimens, chidamide plus thalidomide, or P-GEMOX. Despite the high-risk population, the golidocitinib-based first-line regimen demonstrated promising antitumor efficacy and clinical manageable safety. Further prospective study is warranted to validate the results."

Retrospective data • Cytomegalovirus Infection • Epstein-Barr Virus Infections • Extranodal Natural Killer/T-cell Lymphoma • Febrile Neutropenia • Hematological Malignancies • Infectious Disease • Lymphoma • Neutropenia • Non-Hodgkin’s Lymphoma • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma • Thrombocytopenia • JAK1

Zanubrutinib maintenance post-autologous stem cell transplantation in Mantle Cell Lymphoma: A prospective multicenter Phase II trial (ASH 2025) - P2 | "Additionally, a retrospective analysis used 11 ASCT-treated MCL patients as controls: seven received alternative maintenance regimens (three with anti-CD20 monoclonal antibody; four with thalidomide; one with anti-CD20 antibody combined with lenalidomide; one with ibrutinib who transitioned to anti-CD20 antibody), while four received no maintenance. Conclusion Our preliminary findings indicate that zanubrutinib maintenance demonstrates promising efficacy and a manageable safety profile in post-ASCT MCL patients, supporting its potential as a viable therapeutic option for this population. Keywords Mantle cell lymphoma, autologous hematopoietic stem cell transplantation, maintenance therapy, zanubrutinib"

Clinical • P2 data • Bone Marrow Transplantation • Infectious Disease • Lymphoma • Mantle Cell Lymphoma • Neutropenia • Thrombocytopenia • Transplantation

Developmental Toxicology Profile of the IRAK4 Degrader KT-474. (PubMed, Toxicol Sci) - "Structural similarities of the CRBN-binding portion of these degraders to IMiDs (e.g., thalidomide) have raised safety concerns due to potential degradation of CRBN neosubstrates implicated in teratogenicity, such as SALL4. Deep IRAK4 degradation by KT-474 in primary rat cells, rabbit PBMCs, and a range of tissues provides confidence in the appropriateness of the animal species tested. Taken together, these data clearly differentiate KT-474 from IMiDs, support that CRBN-mediated teratogenicity seen with IMiD drugs is neosubstrate-driven, and demonstrate that structure-based design can generate highly selective degraders devoid of teratogenic risk."

Journal • Targeted Protein Degradation • CRBN • IRAK4 • SALL4

Thalidomide and hydroxyurea combination therapy in transfusion-dependent beta-thalassemia major: A real-world retrospective analysis (ASH 2025) - "Support: None Conflict of Interest Disclosure: The author declares no conflicts of interest. Off-label Disclosure: Thalidomide is used off-label for the treatment of transfusion-dependent beta-thalassemia major."

Combination therapy • Real-world • Real-world evidence • Retrospective data • Beta-Thalassemia • Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders

Thalidomide in beta-thalassemia – a systematic review and meta-analysis (ASH 2025) - "Curative therapies such as allogeneic hematopoietic stem cell transplant and disease-modifying therapies such as Luspatercept have become standard of care. All AEs were grade 1-2 and did not require drug discontinuation. Our results suggest that in settings where gold standard therapies may be unavailable, thalidomide may be a promising treatment option for patients with beta-thalassemia."

Retrospective data • Review • Beta-Thalassemia • Bone Marrow Transplantation • Constipation • Gastroenterology • Gastrointestinal Disorder • Genetic Disorders • Hematological Disorders • Otorhinolaryngology • Vertigo

Evaluating efficacy and safety of hydroxyurea and thalidomide for beta-thalassemia: A systematic review andmeta-analysis. (ASH 2025) - "The treatment regimen consisted of hydroxyurea, thalidomide and aspirin. The results have shown improved haemoglobin levels and reduced ferritin levels, while bilirubin levels showed no significant change. However, given lack of high quality data, further randomised controlled trials are required to validate these results and assess long-term outcomes."

Retrospective data • Review • Beta-Thalassemia • Genetic Disorders

Preliminary results of a first-in-human, Phase 1 study of GLB-002, a novel molecular glue degrader of IKZF1/3, in patients with relapsed or refractory non-Hodgkin lymphoma (ASH 2025) - P1 | "In all 13 enrolled patients with prior treatment of lenalidomide or thalidomide, theORR was 61.5% (8/13, including 5 FL1-3a, 2 LBCL, and 1 marginal zone lymphoma [MZL]). The extent and duration of IKZF1/3 degradation correlated with the dose level andexposure of GLB-002; at 1.0 mg, the maximum degradation of IKZF1/3 was nearly complete.ConclusionGLB-002 monotherapy demonstrated a manageable safety profile and promising antitumor activities inR/R NHL patients, with similar response rate observed in patients who had prior treatment with versuswithout IMiDs, supporting our therapeutic hypothesis that GLB-002, as a next-generation IKZF1/3degrader, can overcome resistance to IMiDs. Enrollment for all NHL subtypes in the expansion cohort isstill ongoing, and further updated data will be presented."

Clinical • First-in-human • P1 data • B Cell Lymphoma • Bone Marrow Transplantation • Follicular Lymphoma • Hematological Malignancies • Indolent Lymphoma • Large B Cell Lymphoma • Lymphoma • Marginal Zone Lymphoma • Non-Hodgkin’s Lymphoma • Targeted Protein Degradation • CRBN • IKZF1 • IKZF3

Real-world comparative outcomes of CAR-T cell therapy versus pomalidomide-based regimens in relapsed/refractory multiple myeloma: A propensity-matched cohort study (ASH 2025) - "Adult RRMM patients (≥18 years) who receivedeither CAR-T therapy without prior exposure to pomalidomide or bispecific antibodies (n=437) orpomalidomide-based regimens without prior CAR-T or bispecifics (n=437) between 2005 and 2025 werepropensity score-matched 1:1.Matching variables included age, sex, race, comorbidities, stem cell transplant status, prior exposure tolenalidomide, bortezomib, carfilzomib, cyclophosphamide, thalidomide, and daratumumab.The index event was the first administration of CAR-T or pomalidomide, with outcomes assessed for 12months following index event. However, CAR-T was associated with higher risks of hospitalization,neutropenia, hypogammaglobulinemia, IVIG use, and markedly increased inflammatory markers,consistent with cytokine release syndrome. These findings highlight the need for vigilant monitoring andsupportive care following CAR-T in RRMM, and suggest that early CAR-T, prior to pomalidomide exposure,may offer survival and..."

CAR T-Cell Therapy • Clinical • IO biomarker • Real-world • Real-world evidence • Chronic Kidney Disease • Cytomegalovirus Infection • Hematological Disorders • Hematological Malignancies • Infectious Disease • Inflammation • Multiple Myeloma • Myelodysplastic Syndrome • Neutropenia • Pneumonia • Renal Disease • Respiratory Diseases • Septic Shock • Thrombocytopenia • IL6

IQGAP1 is a novel non-degraded substrate of CELMoDs and involved in trafficking of immune cells to the tumor microenvironment (ASH 2025) - "Introduction: Cereblon E3 ligase modulating drugs (CELMoDs) represent a novel class ofimmunomodulatory agents that target IKZF1 and IKZF3 for degradation, building upon earliergenerations such as Thalidomide and Avadomide...Similarfindings were observed in patients treated with Golcadomide (Golca), a next generation CELMoDcurrently under clinical investigation for DLBCL, follicular lymphoma, and T cell lymphoma, and wererecapitulated in a genetically engineered humanized cereblon-DLBCL mouse model, highlighting thetranslational significance of CELMoD-induced immune modulation... This study uncovers a novel mechanism by which Golca binding to CRBN mediates enhancedubiquitination of three specific lysine residues on IQGAP1. This modification increased IQGAP1'sinteraction with Rac, CDC42 and actin, thereby promoting directional immune cell migration. Ourfindings provide the first description of IQGAP1 as a non-degraded CELMoD substrate, directly linking itsbiological..."

Biomarker • Immune cell • Tumor microenvironment • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Hematological Malignancies • Immune Modulation • Immunology • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • T Cell Non-Hodgkin Lymphoma • Targeted Protein Degradation • CDC42 • CRBN • IKZF1 • IKZF3 • IQGAP1

The importance of autologous stem cell transplant (ASCT) in multiple myeloma treatment in the lack of novel therapies (ASH 2025) - "Autologous Stem Cell Transplant (ASCT) still is an option for MM patients (pts), especially in big centers with well-establishedroutines for ASCT, and limited access to higher-cost treatment.In Brazil, the public health system (SUS) does not offer access to monoclonal antibodies (mAb) or lenalidomide, and bortezomibhas only recently become available.Objective: This study aims to review the clinical impact and trends of ASCT in a reference public comprehensive cancer center in SouthernBrazil from 2019 to 2024...The group 2019-2021 received CTD (cyclophosphamide,thalidomide, dexamethasone) in 75% of cases...The conditioning regimen was melphalan, Mel200mg/m2 in 71% of pts.The response at D+100 after ASCT were CR:48%, VGPR:37%, PR:5%, Progressive Disease (PD):1%, Ukn:8%... Considering that the Brazilian public health system has limited access to current treatments for MM, ASCT has been establishedin many scenarios, but there is a gap in reporting clinical outcomes.This..."

Hematological Malignancies • Multiple Myeloma • Transplantation

The treatment of patients primary refractory or with sub-optimal response to d-VTD induction: An italian real-life Study (ASH 2025) - "Up tonow in Italy the treatment for newly diagnosed transplant eligible (TE) patients is settled byDaratumumab-Bortezomib-Thalidomide-Dexamethasone (D-VTD) induction; however, there is asignificant proportion of patients who progressed or have a sub-optimal response whose outcome isdismal...As secondline treatment, most patient received KRD as salvage (73/88, 83%), followed by intensive chemo-based(VD/KRD-PACE) regimens in 8 cases (9.1%), other Lenalidomide-based combinations in 4 cases (4.5%) andIsa-KD in 3 cases (3.4%)...The data herein presented confirmed the dismal prognosis of thesefunctional high-risk cases, especially in case of symptomatic and aggressive relapse. However, asignificant proportion of patients can be rescued by the second line treatment, particularly with KRDreinduction and ASCT."

Clinical • Hematological Malignancies • Multiple Myeloma • B2M