Vesanoid (tretinoin) / Roche - LARVOL DELTA

Multicomponent Tandem Reactions for the Synthesis of gem-Dibromo Compounds via an ATRA/Elimination/Nucleophilic Substitution Process. (PubMed, J Org Chem) - "Moreover, this transformation avoids the usage of any catalysts and external additives and also exhibits a broad substrate scope and high functional group compatibility, as demonstrated by the late-stage modification of a pharmaceutically relevant molecule (ibuprofen). Notably, secondary amines play a dual role as bases and nucleophiles in this reaction."

Journal

Efficacy and safety analysis of different treatment regimens in newly diagnosed acute promyelocytic leukemia. (PubMed, Ann Hematol) - "Treatment should be individualized to balance efficacy and complications, with supportive care critical for mitigating infections and bleeding. This study provides clinical evidence for optimizing APL regimens in the chemotherapy-free era."

Journal • Acute Promyelocytic Leukemia • Critical care • Hematological Malignancies • Hepatology • Infectious Disease • Leukemia • Liver Failure • Oncology

Comparison of induction with arsenic trioxide or chemotherapy in a real-world cohort of patients with high-risk acute promyelocytic leukemia. (PubMed, Leukemia) - "Front-line treatment with all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) is superior to standard ATRA and chemotherapy (CHT) in patients with low-/intermediate-risk acute promyelocytic leukemia (APL). The combination of ATRA and ATO was effective and safe in this large, real-world cohort of HR APL patients. The forthcoming results of the APOLLO trial (a direct comparison of ATRA-ATO with ATRA-CHT) might validate our present findings."

Journal • Real-world evidence • Acute Promyelocytic Leukemia • Hematological Malignancies • Leukemia • Oncology

DO APL PATIENTS TREATED WITH ATO+ATRA ACHIEVE A NORMAL LIFE EXPECTANCY? INSIGHTS FROM THE LONG TERM FOLLOW-UP OF THE GIMEMA APL0406 STUDY (EHA 2025) - "Background: Acute Promyelocytic Leukemia (APL) has undergone a paradigm shift in treatment with the introduction of arsenic trioxide (ATO) and all-trans retinoic acid (ATRA), leading to high remission and survival rates. These findings suggest that APL patients achieving remission with ATO+ATRA may experience long-term survival comparable to that of the general population. Given that this is the first study to explore this question, further research with larger cohorts and extended follow-up is warranted to confirm these observations and refine mortality risk estimates."

Clinical • Acute Promyelocytic Leukemia • Hematological Malignancies • Leukemia • Oncology

THE EFFICIENCY AND SAFETY OF LOW-DOSE VENETOCLAX AS A PROPHYLAXIS FOR DIFFERENTIATION SYNDROME IN THE INDUCTION REGIMEN OF ACUTE PROMYELOCYTIC LEUKEMIA (EHA 2025) - "Background: Owing to the induction regimen of all-trans-retinoic acid (ATRA) and arsenic trioxide (ATO), acute promyelocytic leukemia (APL) has become the only type of acute myeloid leukemia (AML) that can be cured without haploidentical hematopoietic stem cell transplantation (haplo-HSCT)... Our study underlined the effective low-dose venetoclax as a prophylaxis of DS during the induction therapy of ATRA and ATO, reducing the early death. No death was observed and the requirement of blood products also get alleviate. Randomized controlled trials with more enrolled patients are needed to be conducted to demonstrate tthe efficiency and safety in future."

Clinical • Acute Kidney Injury • Acute Myelogenous Leukemia • Acute Promyelocytic Leukemia • B Cell Lymphoma • Bone Marrow Transplantation • Hepatology • Infectious Disease • Leukemia • Lymphoma • Nephrology • Oncology • Pneumonia • Pulmonary Disease • Renal Disease • Respiratory Diseases • BCL2

Cu-Catalyzed Electrochemical Atom Transfer Radical Addition with Halomalonic Esters: A Route to Cyclopropane Derivatives. (PubMed, J Org Chem) - "Mechanistic insights into cyclopropanation were elucidated through cyclic voltammetry and UV-vis spectroelectrochemical analysis. This study presents a new mild electrochemical synthetic strategy for constructing functionalized cyclopropane derivatives."

Journal

Chemotherapy without anthracyclines for acute promyelocytic leukemia in a 5-year-old male patient with Fontan procedure. (PubMed, Pediatr Int) - No abstract available

Journal • Acute Promyelocytic Leukemia • Hematological Malignancies • Leukemia • Oncology

Efficacy and Safety of ATO Plus ATRA in Nucleophosmin-1 Mutated Acute Myeloid Leukemia (clinicaltrials.gov) - P1/2 | N=80 | Enrolling by invitation | Sponsor: Institute of Hematology & Blood Diseases Hospital, China | Recruiting ➔ Enrolling by invitation | Trial completion date: Dec 2022 ➔ Oct 2027 | Trial primary completion date: Dec 2021 ➔ May 2026

Enrollment status • Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • NPM1

Long-term Survival of a Lung Transplant Recipient With Acute Promyelocytic Leukemia and Anatomical Variance in the Donor (ATS 2025) - "The patient's immunosuppressive regimen included prednisone, tacrolimus, and mycophenolate mofetil (MMF)...Induction therapy with arsenic trioxide (ATO) and all-trans retinoic acid (ATRA) reduced blast count to 0.4% by day 30...This case highlights a unique instance of long-term survival in a lung transplant recipient with APL who was successfully treated with ATO and ATRA as evidenced by achieving undetectable blast levels. Early and aggressive treatment, along with individualized management, proved essential. Prompt recognition of anatomical variation in the donor's lung and a stepwise approach—such as listing for a re-redo lung transplant when needed—is critical for proactively improving outcomes by addressing potential complications."

Clinical • Acute Promyelocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Leukopenia • Oncology • Otorhinolaryngology • Respiratory Diseases • Transplantation • RARA

All-Trans Retinoic Acid (ATRA) and Atezolizumab for the Treatment of Recurrent or Metastatic Non-Small Cell Lung Cancer (clinicaltrials.gov) - P1 | N=18 | Recruiting | Sponsor: Dwight Owen | Trial completion date: Dec 2024 ➔ Jul 2025 | Trial primary completion date: Dec 2024 ➔ Jul 2025

Trial completion date • Trial primary completion date • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • ALK • PD-L1

Lysari: Lymphocyte-Sparing And Radio-Immunotherapy in Head and Neck Carcinoma (clinicaltrials.gov) - P3 | N=460 | Recruiting | Sponsor: Centre Leon Berard | Not yet recruiting ➔ Recruiting

Enrollment open • Head and Neck Cancer • Hypopharyngeal Cancer • Laryngeal Cancer • Oncology • Oropharyngeal Cancer • Solid Tumor • Squamous Cell Carcinoma

Utilization of RT-PCR and Optical Genome Mapping in Acute Promyelocytic Leukemia with Cryptic PML::RARA Rearrangement: A Case Discussion and Systemic Literature Review. (PubMed, Genes (Basel)) - "This case, in conjunction with the results of our systematic literature review, highlights the importance of performing confirmatory testing in FISH-negative cases of suspected APL to enable prompt diagnosis and appropriate treatment."

Journal • Review • Acute Promyelocytic Leukemia • Hematological Malignancies • Leukemia • Oncology

STARPAC2: A Randomised Trial of ATRA in a Novel Drug Combination for Pancreatic Cancer (clinicaltrials.gov) - P2 | N=170 | Active, not recruiting | Sponsor: Queen Mary University of London | Trial completion date: May 2025 ➔ Feb 2029 | Trial primary completion date: Nov 2024 ➔ Jun 2028

Trial completion date • Trial primary completion date • Hepatology • Oncology • Pancreatic Cancer • Pancreatic Ductal Adenocarcinoma • Solid Tumor

Largest Real-World Evaluation of Treatment Regimens and Clinical Outcomes Among Patients with Acute Promyelocytic Leukemia in the US: Data from the Command Consortium (ASH 2024) - "Chemotherapy-free protocols consisting of arsenic trioxide (ATO) and ATRA have become the standard of care for low-risk APL with high cure rates and reduced hematologic toxicities...At least 1 dose of gemtuzumab ozogamicin was given to 58 (14%) pts, of which 35 (60%) were considered high risk...Outcomes for this disease remain extremely positive, with low 30-day mortality rates noted in this study along with durable OS rates and low incidence of relapse. Additional information on outcomes, safety, and supportive care will be presented at the meeting."

Clinical • Clinical data • HEOR • Real-world • Real-world evidence • Acute Promyelocytic Leukemia • Cardiovascular • Diabetes • Hematological Disorders • Hematological Malignancies • Leukemia • Metabolic Disorders • Oncology • Thrombosis • FLT3 • KRAS • NRAS • WT1

A Study of TAS1440 With ATRA in Subjects With r/r AML (clinicaltrials.gov) - P1 | N=52 | Terminated | Sponsor: Taiho Oncology, Inc. | N=80 ➔ 52 | Trial completion date: Dec 2025 ➔ Feb 2024 | Active, not recruiting ➔ Terminated | Trial primary completion date: Dec 2025 ➔ Feb 2024; The study was terminated due to the changing treatment landscape, difficulty in enrolling eligible participants, and little likelihood of providing meaningful clinical benefit for the participants.

Combination therapy • Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Steric and Distance Effect on Electron Transfer in Dibenzo-Fused BODIPY-Based Photoredox Catalysts. (PubMed, J Phys Chem B) - "Through steady-state and time-resolved optical spectroscopy, electrochemistry, and density functional theory calculations, we elucidated how these simple structural modifications controlled the PeT rates and examined their impacts on catalytic activities in atom transfer radical addition (ATRA) reactions. Our results support previous studies on the (D-A) design of red heavy atom-free photocatalysts."

Journal

Improved Real-World Outcomes of Patients with Acute Promyelocytic Leukemia Treated with First-Line Arsenic Trioxide in Specialized Centers in Portugal through a 24/7 Phone-Based Referral: A Study from the Portuguese Acute Leukemia Group (ASH 2024) - "All patients with genetically confirmed APL who received first-line ATO plus ATRA between January 2017 and May 2024 were included, regardless of disease risk. Despite low numbers, our data support the universal use of DS prophylaxis and first-line ATO's applicability in high-risk patients, who also benefited from this regimen. Through quick initiation of treatment with the effective ATRA + ATO combination, we showed a considerable improvement in the ED rate in 2017-2024, which is a third of the 11.4% ED rate previously reported by our group in a non-high risk APL cohort treated with ATRA + chemotherapy from 2010 to 2017."

Clinical • Real-world • Real-world evidence • Acute Promyelocytic Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Hepatology • Leukemia • Neutropenia • Oncology • Septic Shock

Therapy Based on Attenuated Arsenic Trioxide Plus Low-Dose All-Trans-Retinoic Acid in Acute Promyelocytic Leukemia. Is Less More? (ASH 2024) - P1/2 | "If high-risk (WBC >10 x109/L at presentation) a mitoxantrone 10 mg/m2/day was added on day 1...Differentiation syndrome (DS) prophylaxis with dexamethasone and hydroxyurea or anthracycline was used; supportive transfusion care as needed to reach goals...Despite ATO and ATRA ED remains the main problem in our context. This therapy was effective and safe, even for outpatient use after the late induction phase. This approach is promising, although further follow-up and validation studies are needed to confirm our results."

Acute Kidney Injury • Acute Promyelocytic Leukemia • CNS Disorders • Gastroenterology • Hepatology • Infectious Disease • Metabolic Disorders • Nephrology • Pain • Renal Disease • PML

Prognostic Impact of Complex Karyotype in Acute Promyelocytic Leukemia (ASH 2024) - "Arsenic trioxide (ATO) or chemotherapy plus all-trans retinoic acid (ATRA) frontline treatment regimens are the current standard of care (SOC) for patients with newly diagnosed APL (Sanz et al., 2019)...All patients received treatment, 93 (62.8%) with chemotherapy (idarubicin 12mg/m 2) plus ATRA and 55 (37.2%) with ATO plus ATRA...As therapeutical decisions are based on the initial prognostic risk assessment, the inclusion of CK as a variable in this assessment may provide more precise prognostic information and may indicate a need for better treatment strategies to overcome this adverse effect and improve outcomes in this subgroup of patients. Larger prospective analyses are required to confirm our data."

Acute Myelogenous Leukemia • Acute Promyelocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology

Lysari: Lymphocyte-Sparing And Radio-Immunotherapy in Head and Neck Carcinoma (clinicaltrials.gov) - P3 | N=460 | Not yet recruiting | Sponsor: Centre Leon Berard

New P3 trial • Head and Neck Cancer • Hypopharyngeal Cancer • Laryngeal Cancer • Oncology • Oropharyngeal Cancer • Solid Tumor • Squamous Cell Carcinoma

Standard dose anthracycline plus all-trans retinoic acid and arsenic trioxide as induction chemotherapy significantly reduces early death and relapse for high-risk acute promyelocytic leukemia: a single-center real-world analysis. (PubMed, Ther Adv Hematol) - "All-trans retinoic acid (ATRA) and arsenic trioxide (ATO) have revolutionized the treatment of acute promyelocytic leukemia (APL). WBC ⩾100 × 109/L was correlated with an inferior OS (HR = 3.402, 95% CI: 1.359-8.518). Compared with low-dose anthracycline, standard-dose anthracycline combined with ATO and ATRA dual induction resulted in excellent outcome for high-risk APL patients."

Journal • Real-world • Real-world evidence • Acute Promyelocytic Leukemia • Hematological Malignancies • Leukemia • Oncology

Long-term follow-up of a phase 2 study of all-trans retinoic acid, arsenic trioxide, and gemtuzumab ozogamicin in acute promyelocytic leukemia. (PubMed, Cancer) - P2 | "The combination of ATO-ATRA and GO was curative in 94% of patients who had APL with a favorable safety profile (ClinicalTrials.gov identifier NCT01409161)."

Journal • P2 data • Acute Promyelocytic Leukemia • Hematological Malignancies • Hepatology • Infectious Disease • Leukemia • Oncology

Arsenic trioxide versus Realgar-Indigo naturalis formula in non-high-risk acute promyelocytic leukemia: a multicenter, randomized trial. (PubMed, Haematologica) - P3 | "Following induction therapy with ATRA and ATO, participants were randomly assigned to receive either ATRA plus ATO or ATRA plus RIF both in a 2-week on 2-week off schedule for consolidation therapy. (ClinicalTrials gov. Identifier: as NCT02899169)."

Journal • Acute Promyelocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology

Acute Promyelocytic Leukemia: Long-Term Outcomes from the HARMONY Project. (PubMed, Blood) - "Our study confirms the superiority of ATRA-ATO over ATRA-chemotherapy in APL patients. ED represents an unmet medical need, in particular in older patients and in high-risk APL."

Journal • Acute Myelogenous Leukemia • Acute Promyelocytic Leukemia • Hematological Malignancies • Leukemia • Oncology

Tretinoin and Arsenic Trioxide in Treating Patients With Untreated Acute Promyelocytic Leukemia (clinicaltrials.gov) - P3 | N=158 | Active, not recruiting | Sponsor: Children's Oncology Group | Trial completion date: Sep 2024 ➔ Sep 2025

Trial completion date • Acute Promyelocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3 • PML • RARA