Microscopic view of platelets supported by Vitamin A and androgen symbols, representing ITP treatment with ATRA and Danazol.

Beating ITP: A New Combo Therapy Shows Promise

Lena Voss in Health & Wellness December 2025 • 5 min read.

"All-trans retinoic acid (ATRA) plus danazol offers renewed hope for adults battling primary immune thrombocytopenia (ITP) when other treatments fail."

Primary immune thrombocytopenia (ITP) is a serious autoimmune disorder where the body mistakenly attacks and destroys its own platelets, which are essential for blood clotting. This can lead to increased bleeding and bruising, significantly impacting a person's quality of life. While initial treatments are often effective, many adults with ITP find that these therapies eventually fail, leaving them searching for alternative options.

Traditional second-line treatments for ITP include splenectomy (surgical removal of the spleen), rituximab, and thrombopoietin receptor agonists. However, these treatments come with their own set of limitations and potential side effects. Researchers have been exploring new approaches, and one promising avenue involves the use of all-trans retinoic acid (ATRA), a derivative of vitamin A known for its immunomodulatory effects.

A recent multi-center, randomized, phase 2 trial investigated the efficacy and safety of combining ATRA with danazol, an attenuated androgen, in adults with corticosteroid-resistant or relapsed ITP. The results of this study offer new hope for individuals seeking more effective and sustained remission from ITP.

ATRA Plus Danazol: A Powerful Combination for ITP

Microscopic view of platelets supported by Vitamin A and androgen symbols, representing ITP treatment with ATRA and Danazol.

The study, conducted across five medical centers in China, involved 96 adult patients with ITP who had not responded to or had relapsed after corticosteroid treatment. Participants were randomly assigned to receive either ATRA plus danazol or danazol alone. The primary goal was to assess the 12-month sustained response rate – defined as a platelet count of at least 30 × 109 per L (with a doubling of baseline) or a platelet count of at least 100 × 109 per L, without bleeding or needing rescue medication.

The results were compelling. Patients receiving the combination therapy of ATRA plus danazol experienced a significantly higher sustained response rate compared to those receiving danazol alone.

62% of patients in the ATRA plus danazol group achieved a sustained response, compared to only 25% in the danazol group.
Patients in the combination therapy group also experienced a more rapid initial response, with a target platelet count achieved sooner than those in the danazol group.
Importantly, the combination therapy was well-tolerated, with only a few mild adverse events reported.

These findings suggest that ATRA, when combined with danazol, can be a more effective second-line treatment option for adults with ITP who have not responded to corticosteroids. The combination appears to offer a synergistic effect, leading to improved platelet counts and reduced bleeding risk.

Looking Ahead: New Hope for ITP Patients

The study's findings provide a promising avenue for ITP patients seeking effective second-line treatments. While further research is needed to determine the optimal dosage and long-term effects of ATRA in combination with danazol, this study offers a compelling case for its use in patients who have not responded to standard therapies.

The combination of ATRA and danazol represents a potential shift in the treatment paradigm for ITP, offering a less invasive and more targeted approach to managing the condition. This could translate to a better quality of life for individuals living with ITP, allowing them to live more active and fulfilling lives.

For those struggling with ITP and seeking alternative treatment options, it's important to discuss these findings with a hematologist or healthcare provider. They can assess individual circumstances and determine whether ATRA plus danazol is a suitable treatment option.

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This article is based on research published under:

DOI-LINK: 10.1016/s2352-3026(17)30170-9, Alternate LINK

Title: Oral All-Trans Retinoic Acid Plus Danazol Versus Danazol As Second-Line Treatment In Adults With Primary Immune Thrombocytopenia: A Multicentre, Randomised, Open-Label, Phase 2 Trial

Subject: Hematology

Journal: The Lancet Haematology

Publisher: Elsevier BV

Authors: Fei-Er Feng, Ru Feng, Min Wang, Jia-Min Zhang, Hao Jiang, Qian Jiang, Jin Lu, Hui Liu, Jun Peng, Ming Hou, Jian-Liang Shen, Jing-Wen Wang, Lan-Ping Xu, Kai-Yan Liu, Xiao-Jun Huang, Xiao-Hui Zhang

Published: 2017-10-01

Everything You Need To Know

What exactly is Primary Immune Thrombocytopenia (ITP), and why is it so important to find effective treatments?

Primary Immune Thrombocytopenia, or ITP, is an autoimmune disorder where the body's immune system mistakenly attacks and destroys platelets. Platelets are essential for blood clotting, so this destruction can lead to increased bleeding and bruising. The impact of ITP on a person's life can be significant, and finding effective treatments is crucial for improving their quality of life.

What was the main focus of this study regarding the treatment of ITP?

The study focused on a combination therapy of All-trans retinoic acid, also known as ATRA, and danazol. This combination was given to adult patients with ITP who had not responded to corticosteroid treatment. The goal was to see if this combination could lead to a sustained increase in platelet count, reducing bleeding and improving overall well-being. The success of ATRA and danazol suggests a synergistic effect, enhancing the body's ability to produce and maintain healthy platelet levels.

What were the key results of the study regarding the effectiveness of ATRA plus danazol compared to danazol alone?

The study showed that 62% of patients receiving the combination therapy of ATRA plus danazol achieved a sustained response, compared to only 25% in the danazol-only group. This means that the combination was significantly more effective in increasing platelet counts and reducing bleeding risk in those who had not responded to corticosteroids. The results highlight the potential of ATRA in combination with danazol as a more effective second-line treatment option.

Besides ATRA and danazol, what other treatment options are available for ITP, and how do they compare?

While the study showed promising results for ATRA and danazol, it is important to remember that traditional second-line treatments like splenectomy, rituximab, and thrombopoietin receptor agonists are still relevant. These treatments have been used for years and can be effective for some patients. Further research is needed to determine when ATRA and danazol should be used in relation to these established options, and what factors might predict which patients will respond best to each treatment approach.

What is All-trans retinoic acid (ATRA), and how does it work to help people with ITP?

All-trans retinoic acid, or ATRA, is a derivative of vitamin A that is known for its immunomodulatory effects. In the context of ITP, it is believed that ATRA helps to regulate the immune system and prevent it from attacking platelets. By modulating the immune response, ATRA can help to increase platelet counts and reduce bleeding risk. The specific mechanisms by which ATRA exerts its effects in ITP are still being investigated, but its immunomodulatory properties make it a promising therapeutic agent.