Chronic myeloid leukemia (CML) blast crisis
About
CML is driven by the BCR-ABL fusion gene, and the chronic phase can be effectively controlled by tyrosine kinase inhibitors (TKIs). However, blast crisis stage (BP-CML) is resistant to TKI and has a very poor prognosis. Core dilemmas: ① There is widespread resistance to existing TKIs during the acute crisis period;② Transplantation needs to be returned to the chronic period;③ Allogeneic transplantation is the only way to cure the disease, but the transplant-related mortality rate is high.
Features & Symptoms
- Chronic stage progresses to acute crisis stage
- Resistance to tyrosine kinase inhibitors
- Similar to acute leukemia
- very poor prognosis
Treatment Options
| Treatment | Description | Stage | Price Range |
|---|---|---|---|
| Ponatinib in combination with chemotherapy | The third-generation TKI, prnitinib, is effective against T315I mutations, and is combined with chemotherapy to achieve transplantation after returning to the chronic stage. | FDA批准 | $200K - $500K/年 |
| Asciminib (STAMP inhibitor) | The allosteric inhibitor Asciminib acts on the myristoyl pocket of BCR-ABL and is effective against multiple drug-resistant mutations. | FDA批准 | $250K - $600K/年 |
| allogeneic hematopoietic stem cell transplantation | Patients in acute crisis need to return to the chronic stage through treatment before undergoing allogeneic transplantation. | 临床应用 | $300K - $800K/次 |
Prognosis
Median survival time in the crisis period is 6-12 months
Additional Notes
Notes: Mutation detection in BCR-ABL kinase region: Mutation detection should be carried out as soon as possible during the blast crisis period, and patients with T315I mutation can choose platinib; treatment goal: During the blast crisis period, we should strive to return to the chronic phase as soon as possible (Through strong chemotherapy or TKI), transplant indications should be evaluated as soon as possible after returning to the chronic phase;TKI selection: Pronatinib (third-generation TKI) is effective against most BCR-ABL mutations, but the risk of vascular events should be paid attention to; Transplantation evaluation: Allogeneic hematopoietic stem cell transplantation should be evaluated as soon as possible after returning to the chronic phase. Transplantation is currently the only possible method to cure CML in blast crisis.; Exclusive service commitment: The platform will allocate a dedicated medical team to each patient to assist in dismantling and properly deploying the following precautions throughout the process. When cross-state and cross-border diagnosis and treatment projects or drugs are involved, the platform will make overall arrangements for the entire docking; for disabled and semi-disabled patient groups, the platform will provide rush-free alternative solutions (including remote consultation, door-to-door sampling, direct drug delivery in the cold chain, etc.), to ensure that patients can get the same high-quality diagnosis and treatment resources without having to travel in person.; Study phase: FDA approval. Prognosis: The median survival time in acute crisis is 6-12 months.
Frequently Asked Questions
What is Chronic myeloid leukemia (CML) blast crisis?
CML is driven by the BCR-ABL fusion gene, and the chronic phase can be effectively controlled by tyrosine kinase inhibitors (TKIs). However, blast crisis stage (BP-CML) is resistant to TKI and has a very poor prognosis. Core dilemmas: ① There is widespread resistance to existing TKIs during the acute crisis period;② Transplantation needs to be returned to the chronic period;③ Allogeneic transplantation is the only way to cure the disease, but the transplant-related mortality rate is high.
What are the symptoms of Chronic myeloid leukemia (CML) blast crisis?
Chronic stage progresses to acute crisis stage,Resistance to tyrosine kinase inhibitors,Similar to acute leukemia,very poor prognosis
How is Chronic myeloid leukemia (CML) blast crisis treated?
Ponatinib in combination with chemotherapy: The third-generation TKI, prnitinib, is effective against T315I mutations, and is combined with chemotherapy to achieve transplantation after returning to the chronic stage.; Asciminib (STAMP inhibitor): The allosteric inhibitor Asciminib acts on the myristoyl pocket of BCR-ABL and is effective against multiple drug-resistant mutations.; allogeneic hematopoietic stem cell transplantation: Patients in acute crisis need to return to the chronic stage through treatment before undergoing allogeneic transplantation.
What is the prognosis for Chronic myeloid leukemia (CML) blast crisis?
Median survival time in the crisis period is 6-12 months