Childhood Acute Promyelocytic Leukemia
Conditions
Brief summary
Outcome of acute promyelocytic leukemia (APL) has greatly improved since the introduction of all-trans-retinoic acid (ATRA). Treatment with ATRA and anthracycline-based chemotherapy (ATRA + chemotherapy) decreases relapses of the disease as well as early hemorrhagic deaths. Nowadays patients with APL have an event-free survival (EFS) of up to 80%. However, there remains a subset of the patients in whom the disease relapses. Recently, a randomized prospective study showed that the addition of ATO to ATRA + chemotherapy treatment protocol had a significantly higher EFS in patients with APL than those treated with ATRA + chemotherapy protocol. The patients treated with ATO + ATRA + chemotherapy had a five years EFS of 89.2%. Moreover, a recent study showed that Indigo naturalis formula (RIF), a traditional Chinese medicine with tetraarsenic tetrasulfide (As4S4), indirubin, and tanshinone IIA as major active ingredients, yielded synergy in the treatment of a murine APL model in vivo and in the induction of APL cell differentiation in vitro . It is about 20 years since RIF was used to treat ALP in China. Clinical studies showed that this agent was effective against APL. Compared to ATO, RIF is relatively inexpensive and can be taken orally, resulting in reducing the number of hospital days and the treatment cost. However, there is no report comparing treatment outcomes of ATO + ATRA + chemotherapy and RIF + ATRA + chemotherapy protocols in children with APL so far. For this purpose, therefore, investigators are going to conduct a multicenter and randomized prospective study in children with APL.
Detailed description
OBJECTIVES: * Determine the safety and efficacy of ATO/RIF + ATRA + less intensive chemotherapy protocol in children with acute promyelocytic leukemia (APL). * Compare the safety，efficacy and treatment cost of RIF + ATRA + less intensive chemotherapy with ATO + ATRA + less intensive chemotherapy protocol in children with APL. Determine if ATO can be substituted by RIF. OUTLINE: This is a multicenter and randomized prospective study. PROJECTED ACCRUAL: A total of 162 patients will be accrued for this study.
Interventions
Sponsors
South China Children's Leukemia Group
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 16 Years
Healthy volunteers
No
Inclusion criteria
* Patients less than 16 years old with newly diagnosed PML-RARa positive acute promyelocytic leukemia.
Exclusion criteria
* Patients who have coma, convulsion or paralysis due to intracranial hemorrhage or central nervous system leukemia at diagnosis.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| event-free survival | 5 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| hospitalization cost | 2 years | The cost mainly includes the fees of hospital bed, drugs, therapies and blood products. Time frame: from the beginning of induction therapy to the end of maintenance treatment. |
Countries
China
Outcome results
None listed