Professor Sun Zhiqiang: The fire of innovation, casting the blade of treatment, from the real drug use of Rotxip and core research β- the future treatment trend of local poverty

For the treatment of β-thalassemia (hereinafter referred to as "β-ground poverty"), the previous means were more limited

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Doctor of Medicine, Chief Physician, Master Supervisor

Director of the Department of Hematology, Shenzhen Hospital, Southern Medical University

• Member of the Hematology Branch of Guangdong Medical Association

• Member of the Hematologist Branch of Guangdong Medical Doctor Association

• Member of the Blood Immunology Branch of the Chinese Society of Immunology

• Vice Chairman of the Blood Branch of the Rehabilitation Association of the Chinese Anti-Cancer Association

• Chairman of the Lymphoma Professional Committee of Shenzhen Anti-Cancer Association

• Vice Chairman of Hematologist Branch of Shenzhen Medical Doctor Association

• Vice Chairman of Lymphoma Branch of Shenzhen Medical Doctor Association

• Vice Chairman of leukemia Committee of Shenzhen Anti-Cancer Association

• Vice Chairman of Shenzhen Lymphoma Branch of Anti-Cancer Alliance

• Vice Chairman of the Cellular Immunology Professional Committee of Shenzhen Health Management Association

• Member of the Standing Committee of the Hematology Branch of Shenzhen Medical Association

• Member of the Standing Committee of the Hematology and Oncology Branch of the Guangdong Preventive Medicine Association

• Member of the Standing Committee of the Precision Medicine Association of Shenzhen Anti-Cancer Association

• Member of the Standing Committee of the Hematology and Tumor Branch of Shenzhen Anti-Cancer Association

• Member of the Oncology and Microecology Committee of the Chinese Anti-Cancer Association

"The patient is 20 years old, needs long-term blood transfusion, has had fever, chills and other transfusion reactions and fears blood transfusion, so the transfusion compliance is poor, and standardized treatment cannot be adhered to; In addition, due to long-term blood transfusions, excess iron deposition in various organs such as the heart and liver causes damage to related organs, and patients have adverse reactions to the digestive tract such as decreased appetite, diarrhea, etc.

The clinical application of Rotxip after marketing has attracted clinical attention, because the first-line clinical response often represents the most intuitive feelings

Before rotsipp was listed, the experience of patients in Shenzhen Hospital may be a true portrayal
of the vast number of β in China - poor patients.

Although hematopoietic stem cell transplantation is the only means that can cure the disease, there are problems such as
age restrictions and low success rate of matching.

Severe or transfusion-dependent adult patients who are unable to receive transplantation often require a lifelong standard of blood transfusion and iron removal therapy
.

Many limitations, such as iron overload caused by long-term blood transfusions, blood supply tension in some areas, which makes it difficult to guarantee blood transfusion treatment, poor treatment compliance, and gradually increasing economic pressure, have brought heavy burdens
to β-poor patients and society.

In view of the diagnosis and treatment of β-local poverty, Professor Sun Zhiqiang pointed out, "The clinical demand for innovative drugs is urgent
.

Rotxip can target the ineffective hematopoiesis of β-ground poverty, selectively combine with the TGF-β superfamily ligand, a key factor that regulates red blood cell maturation, reduce the abnormally enhanced Smad 2/3 signaling pathway transduction, thereby promoting advanced red blood cell maturation, enabling the body to produce more mature red blood cells, increasing hemoglobin levels, reducing the patient's transfusion burden and dependence on iron removal therapy, and improving treatment adherence
.

”

The premise that innovative drugs can be clinically applied is that there is definite evidence of evidence-based medicine
.

The BELIEVE study has confirmed that rotsiep significantly improves transfusion dependence in transfusion-dependent β-poor adult patients
.

The results presented at this year's Annual Meeting of the European Association of Hematology (EHA) confirm the efficacy and safety
of long-term treatment of Rotsheep.

• Persistent decrease in the transfusion burden of patients: with a median treatment of about 154 weeks, the burden of transfusion in the Rotsipp group decreased from baseline≥ and the proportion of patients with 33% at any 12 and 24 weeks was 77.
  2% and 51.
  8%, respectively, compared with 70.
  5% and 41.
  1% at the median treatment of 64 weeks; Similar results were seen in the proportion of patients with a lower transfusion burden than baseline≥ 50 percent of patients
  .
  
  

• More patients were released from erythrocyte infusion ≥ 8 weeks: at about 154 weeks of median treatment, the proportion of patients in the Rotsiep group who were out of red blood cell infusion ≥ 8 weeks was 12.
  1%, higher than the median treatment at 64 weeks of 10.
  7%.
  
  
  The median (95% CI) maximum duration was 72.
  0 (62.
  0 to 103.
  0) days
  .
  

• Hepatic iron concentrations (LIC) decreased in responding patients: in any 12-week ≥33% of patients with a decrease in transfusion burden from baseline, the mean LIC decreased by 3.
  73 mg/g dry weight
  from baseline at 144 weeks.
  
  

• Patient response duration was prolonged with continued rothexip: at about 154 weeks of median treatment versus 64 weeks, during any 12 weeks, the transfusion burden of rothesipre-treated patients decreased from baseline by ≥ 33% The median maximum duration was extended from 104 days to 114 days, with an average overall (cumulative) duration of up to 627 days
  .
  
  

• Rottersip is well tolerated for long-term treatment, and no new safety issues have been reported; No malignant hematological tumors were observed in the Rotsipp group during the study period, nor were precancerous lesions
  reported.
  
  

The significance of the publication of the results of the BELIEVE heavy long-term follow-up study is self-evident, which not only helps to enhance confidence in the application of Rotsipp in clinical treatment, but also hopes to have a positive impact on
society at the macro level.

In this regard, Professor Sun Zhiqiang said, "It has been estimated that the annual blood transfusion volume of Chinese patients who respond to Rotxip treatment is expected to be reduced by about 57%, and the number of blood transfusions is reduced by about 21%.


In addition, patients improved some health-related quality of life scores, helping to improve tolerance to treatment
.

In addition, Rotsipp helps to reduce the economic burden of blood transfusions and complication treatment, increase productivity, reduce the cost of complication treatment, and it is estimated that patients who respond to Rotsiep treatment will reduce the miss time due to blood transfusion-related problems by about 78% and increase productivity by about 26% throughout the year, which provides a boost for young adult patients to re-enter society and increases their sense
of social identity.

”

The key to the clinical role of innovative drugs is to promote standardized treatment
.

Looking internationally, Rothipp has been recommended by the International Federation of Poor Ground (TIF) guidelines for the treatment
of transfusion-dependent β-poor in adults.

Based on China, Professor Sun Zhiqiang said: "The "Standardized Diagnosis and Treatment of Thalassemia" project, which is participated by the top experts in Chinese hematology, has been launched in 2021; At present, the Hematology Branch of the Chinese Medical Association is also formulating the "Guidelines for the Diagnosis and Treatment of Blood Transfusion-Dependent β Thalassemia in China (2022 Edition)" to promote the standardization of clinical treatment of patients with β-ground poverty, explore the clinical application of innovative drugs, and improve the level of
clinical diagnosis and treatment.

”

Improving drug accessibility is the only way for more patients to truly enjoy the efficacy and safety of new drugs
.

In this regard, Professor Sun Zhiqiang said: "On the one hand, we should strengthen the education of doctors and patients, and improve the awareness of diseases and innovative drugs; On the other hand, we should promote Rotxip to enter medical insurance as soon as possible to meet the needs of the majority of patients and enable more patients with β-poor to benefit
.

Doctors, patients, and social support systems should work together to escort β-poor patients throughout the process
.

”

After more than ten years, a new dawn
finally came.

The listing of the innovative drug Rotsipp has broken the dilemma of no new drugs being approved in the field of domestic β-ground poverty treatment for a long time, and is expected to open a new era to help patients have more confidence in receiving clinical treatment and improve the quality of life of
patients.

It is believed that at that time, after the further improvement of the standardized diagnosis and treatment system and the further improvement of the accessibility of drugs, China's β-poor patients will eventually clear the obstacles to treatment and gain a new life
.

References:

1.
Maria Domenica Cappellini, et al.
, 2022 EHA, S270.

2.
Vip Viprakasit, et al.
, 2022 EHA, P1518

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