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acute promyelocytic leukemia - Clinical Profile, Treatments & Trials | Kisho | Kisho

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

1 FDA-approved treatment is available for acute promyelocytic leukemia, including ARSENIC TRIOXIDE (TRISENOX, approved 2000). An additional 4 compounds hold orphan drug designation.

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved	Market Status
TRISENOX	ARSENIC TRIOXIDE	—	2000	Available

Orphan Drug Designations

The following drugs have received orphan drug designation from the FDA for acute promyelocytic leukemia. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor	Designated	Exclusivity End	Designation Status
menatetrenone	menatetrenone	NBI Pharmaceuticals, Inc.	2010	—	Designated
Tamibarotene	Tamibarotene	CytRx Corporation	2007	—	Designated
Phenylbutyrate	Phenylbutyrate

Gene therapy approaches for acute promyelocytic leukemia have been reported in the published literature.

Clinical Trials

16 trials found

Explore Trials

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

16 clinical trials registered, 6 recruiting. Interventions under study include drug therapy, other interventions, and procedural interventions. Pipeline includes 3 PHASE3, 6 PHASE2, 1 PHASE1. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT04687176](https://clinicaltrials.gov/study/NCT04687176)	Frontline Oral Arsenic Trioxide for APL	PHASE2	The University of Hong Kong	RECRUITING
[NCT04793919](https://clinicaltrials.gov/study/NCT04793919)	Treatment Study for Children and Adolescents With Acute Promyelocytic Leukemia	PHASE2	Associazione Italiana Ematologia Oncologia Pediatrica	RECRUITING
[NCT04897490](https://clinicaltrials.gov/study/NCT04897490)	RWE of 1st Line Treatment With ATO/ATRA for Adult APL	—	Grupo Argentino de Tratamiento de la Leucemia Aguda	RECRUITING
[NCT07504458](https://clinicaltrials.gov/study/NCT07504458)	Pivotal Open-label Phase 3 Clinical Study of QTX-2101 in Adult Patients With Acute Promyelocytic Leukemia	PHASE3	Quetzal Therapeutics	RECRUITING
[NCT01409161](https://clinicaltrials.gov/study/NCT01409161)	Tretinoin and Arsenic Trioxide With or Without Gemtuzumab Ozogamicin in Treating Patients With Previously Untreated Acute Promyelocytic Leukemia	PHASE2	M.D. Anderson Cancer Center	RECRUITING

201 publications have been identified in PubMed for acute promyelocytic leukemia. Research spans Basic Science / Preclinical (24%), Review / Meta-Analysis (22%), and Clinical Trial Publication (15%).

Research Type	Count	% of Total
Laboratory research	48	24%
Research summaries	43	22%
Clinical study results	29	15%
Patient case studies	27	14%
Disease patterns and progression	26	13%
Testing and diagnosis research	11

Recent Publications

Ahmad H (2026). [PMID: 41906112](https://pubmed.ncbi.nlm.nih.gov/41906112/). *BMC Cancer*. [Review / Meta-Analysis]
Reikvam H (2026). [PMID: 42095625](https://pubmed.ncbi.nlm.nih.gov/42095625/). *Expert Rev Hematol*. [Other]
Dapueto G (2026). [PMID: 41389189](https://pubmed.ncbi.nlm.nih.gov/41389189/). *Expert Rev Anticancer Ther*. [Diagnostic / Biomarker]
Rodríguez-Veiga R (2026). [PMID: 41230704](https://pubmed.ncbi.nlm.nih.gov/41230704/). *Haematologica*. [Clinical Trial Publication]
Lee JH (2026). [PMID: 41670515](https://pubmed.ncbi.nlm.nih.gov/41670515/). *Clin Lab*. [Case Report / Case Series]
Kamat G (2026). [PMID: 42040735](https://pubmed.ncbi.nlm.nih.gov/42040735/). *Indian J Hematol Blood Transfus*. [Epidemiology / Natural History]
Takahashi S (2026). [PMID: 41824616](https://pubmed.ncbi.nlm.nih.gov/41824616/). *Acta haematologica*. [Basic Science / Preclinical]
Zong S (2026). [PMID: 41513364](https://pubmed.ncbi.nlm.nih.gov/41513364/). *Anal Chim Acta*. [Diagnostic / Biomarker]
Yoham AL (2026). [PMID: 32491410](https://pubmed.ncbi.nlm.nih.gov/32491410/). *Unknown Journal*. [Gene Therapy / Novel Therapeutics]
Marvin-Peek J (2026). [PMID: 41910474](https://pubmed.ncbi.nlm.nih.gov/41910474/). *Expert Rev Clin Pharmacol*. [Review / Meta-Analysis]

Kisho

acute promyelocytic leukemia

Overview

Clinical Presentation

Explore clinical features

Diagnosis

Treatment

Approved Therapies

Orphan Drug Designations

Clinical Trials

Prognosis

Research Landscape

Recent Publications

Medical References

External Resources

OMIM

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

acute promyelocytic leukemia

Overview

Clinical Presentation

Explore clinical features

Diagnosis

Treatment

Approved Therapies

Orphan Drug Designations

Clinical Trials

Prognosis

Research Landscape

Recent Publications

Medical References

External Resources

OMIM

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team