TriangleHEALTH PROFESSIONAL · SOURCE READING
HSCT
Source: Childhood Acute Promyelocytic Leukemia Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: June 14, 2024 · Captured 2026-09-09.
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Context: Treatment of Recurrent APL
Retrospective pediatric studies have reported 5-year event-free survival (EFS) rates after either autologous or allogeneic transplant approaches to be similar, at approximately 70%.[20,21]
Evidence (autologous HSCT):
A study in adult patients treated with an autologous transplant demonstrated the following:[22]
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A study in adult patients treated with an autologous transplant demonstrated the following:[22]
There was an improved 7-year EFS rate (77% vs. 50%) when both the patient and the stem cell product had negative PML::RARA fusion transcripts by polymerase chain reaction (molecular remission) before transplant.
Another study demonstrated that among seven patients undergoing autologous HSCT and whose cells were minimal residual disease (MRD) positive, all relapsed in less than 9 months after transplant. However, only one of eight patients whose autologous donor cells were MRD negative relapsed.[23]
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An additional report demonstrated a difference in survival based on the treatment received during relapse.[24]
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An additional report demonstrated a difference in survival based on the treatment received during relapse.[24]
The 5-year EFS rate was 83.3% for patients who underwent autologous HSCT in second molecular remission.
An additional report demonstrated a difference in survival based on the treatment received during relapse.[24]
The 5-year EFS rate was 34.5% for patients who received only maintenance therapy.
Another retrospective report found an improved survival for patients treated with HSCT after achieving a molecular remission.[13]
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Another retrospective report found an improved survival for patients treated with HSCT after achieving a molecular remission.[13]
Ninety-four percent of pediatric and adult patients (64 of 67) with relapsed APL, after primarily receiving single-agent arsenic trioxide, achieved a molecular remission after treatment with arsenic-containing reinduction regimens.
Another retrospective report found an improved survival for patients treated with HSCT after achieving a molecular remission.[13]
For patients who received postremission consolidation with HSCT (n = 35), the 5-year overall survival (OS) rate was 90.3% (± 5.3%), and the EFS rate was 87.1% (± 6.0%). These outcomes were significantly superior to the outcomes of patients who received an arsenic-containing maintenance regimen, which resulted in a 5-year OS rate of 58.6% (± 10.4%) and an EFS rate of 47.7% (± 10.3%).
Such data support the use of autologous transplant in patients who are MRD negative in second complete remission and have MRD-negative stem cell collections.
Because of the rarity of APL in children and the favorable outcome for this disease, clinical trials in relapsed APL to compare treatment approaches are likely not feasible. However, an international expert panel provided recommendations for the treatment of relapsed APL on the basis of the reported pediatric and adult experiences.[25]
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Publication references
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Preserved source evidence · Not medical advice