Vesanoid (tretinoin) / Roche - LARVOL DELTA

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

Different prognosis according to treatment in patients with acute promyelocytic leukemia: How the outcome changed over time. (PubMed, Ann Hematol) - "Complete response (CR) was achieved in 97.4%, 100%, 100%, and 27% of patients treated with AIDA protocol, AIDA + Ara-C, ATRA + ATO, and ATRA monotherapy, respectively. Among low and intermediate-risk patients, age > 70 years (p = 0.027) and relapse (p < 0.001) were significantly associated with reduced outcomes. This study contributes to the advancement of our understanding of APL treatment, underscoring the ongoing need for research to enhance outcomes and explore new therapeutic approaches and prognostic factors."

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

Response Rates and Transplantation Impact in Patients with Relapsed Acute Promyelocytic Leukemia. (PubMed, Cancers (Basel)) - "Our findings support the efficacy of ATO-based therapies in first relapse of APL and confirm the achievement of molecular remission as an independent outcome predictor in both first and second APL relapse."

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

16-1080.cc: Pembrolizumab and All-Trans Retinoic Acid Combination Treatment of Advanced Melanoma (clinicaltrials.gov) - P1/2 | N=26 | Completed | Sponsor: University of Colorado, Denver | Active, not recruiting ➔ Completed

Trial completion • Melanoma • Oncology • Solid Tumor

ARSENIC TRIOXIDE-INDUCED PERIPHERAL NEUROPATHY: PROSPECTIVE CLINICAL AND NEUROPHYSIOLOGICAL EVALUATION IN A COHORT OF PATIENTS WITH ACUTE PROMYELOCYTIC LEUKEMIA (SIE 2024) - "While neurotoxicity is a well-known effect associated with arsenic poisoning, data regarding patients being treated with the ATO/ATRA regimen is scarce. Conclusions. Although almost half of the enrolled subjects in our study manifested clinical and/or neurophysiological evidence of sensory axonal PN during ATO treatment, such toxicity seems to be reversible, with clinical and neurophysiological improvement being documented at follow-up evaluations, and only a minority of patients still being symptomatic after treatment interruption."

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

Leucocytosis during induction therapy with all-trans-retinoic acid and arsenic trioxide in acute promyelocytic leukaemia predicts differentiation syndrome and treatment-related complications. (PubMed, Br J Haematol) - "All cases were successfully managed with hydroxyurea...In conclusion, APL patients undergoing ATRA-ATO therapy with lower fibrinogen levels and platelet counts at diagnosis and with a massive bone marrow blast infiltrate should be carefully monitored for the development of leucocytosis during induction. DS and other treatment-related complications seem to occur almost exclusively in patients developing leucocytosis, who should necessarily receive DS prophylaxis and more intensive monitoring and supportive therapy to prevent treatment complications."

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

Ischemic Stroke as a Presenting Feature of Promyelocytic Blast Phase in Chronic Myeloid Leukemia – an Uncommon Presentation of an Exceptionally Rare Blastic Transformation of Myeloproliferative Neoplasm (SOHO 2024) - "positivity was started on imatinib (400 mg) after which she reported significant symptomatic relief...Due to significant risk concerning QT prolongation, dasatinib was switched to bosutinib without altering ATO and ATRA... This case report characterizes an extremely unusual presentation of the promyelocytic blast phase in CML with the initial manifestation of coagulopathy in thrombosis. Early initiation of therapy helped the patient to survive this aggressive leukemia, highlighting the importance of morphological assessment despite the atypical and not-so-classical immunophenotyping findings."

Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Myeloproliferative Neoplasm • Oncology • ABL1

Nivolumab and All-trans Retinoic Acid for Pancreatic Cancer (clinicaltrials.gov) - P1 | N=10 | Active, not recruiting | Sponsor: China Medical University Hospital | Recruiting ➔ Active, not recruiting | N=20 ➔ 10

Enrollment change • Enrollment closed • Gastrointestinal Cancer • Hepatology • Oncology • Pancreatic Cancer • Solid Tumor

Serial Measurements of Molecular and Architectural Responses to Therapy (SMMART) PRIME Trial (clinicaltrials.gov) - P1 | N=2 | Terminated | Sponsor: OHSU Knight Cancer Institute | Active, not recruiting ➔ Terminated; Low accrual

Trial termination • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Anemia • Breast Cancer • Castration-Resistant Prostate Cancer • Chronic Lymphocytic Leukemia • Chronic Myeloid Leukemia • Chronic Myelomonocytic Leukemia • Gastrointestinal Cancer • Genito-urinary Cancer • Hematological Disorders • Hematological Malignancies • Hepatology • Hodgkin Lymphoma • Leukemia • Lymphoma • Multiple Myeloma • Myelodysplastic Syndrome • Myelofibrosis • Myeloproliferative Neoplasm • Non-Hodgkin’s Lymphoma • Oncology • Pancreatic Adenocarcinoma • Pancreatic Cancer • Prostate Cancer • Small Lymphocytic Lymphoma • Solid Tumor • T Acute Lymphoblastic Leukemia • Thrombocytosis

VENETOCLAX AND SORAFENIB ARE EFFECTIVE IN RELAPSED ACUTE PROMYELOCYTIC LEUKEMIA WITH REFRACTORY CENTRAL NERVOUS SYSTEM INVOLVEMENT (EHA 2024) - "ATRA, arsenic trioxide (ATO), bortezomib are effective intreatment of APL relapse but do not penetrate blood-brain barrier...Triple intrathecal therapy with the administration of methotrexate (MTX),cytarabine (AraC) and dexamethasone (DEX) was performed on days 6, 8, 13 only, as well as venetoclax 400mg/day orally continuously started on day 9 and sorafenib 400 mg 2 times a day orally continuously started onday 16...The cycle of induction therapy for bonemarrow relapsed APL started according to the ATO+ATRA+bortezomib protocol... This case demonstrate the effectiveness of venetoclax and sorafenib in the treatment of chemotherapyrefractory CNS involvement in relapsed APL patient. The possible differentiating effect observed duringvenetoclax and sorafenib treatment, with the appearance of a more mature cell population in the CSF alongwith the blast population with the subsequent disappearance of both."

Acute Promyelocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • CD14 • CD33 • CD34 • CD7 • KIT • NCAM1 • PTPRC