211 research outputs found
Detection of minimal residual disease identifies differences in treatment response between T-ALL and precursor B-ALL
We performed sensitive polymerase chain reaction-based minimal residual
disease (MRD) analyses on bone marrow samples at 9 follow-up time points
in 71 children with T-lineage acute lymphoblastic leukemia (T-ALL) and
compared the results with the precursor B-lineage ALL (B-ALL) results (n =
210) of our previous study. At the first 5 follow-up time points, the
frequency of MRD-positive patients and the MRD levels were higher in T-ALL
than in precursor-B-ALL, reflecting the more frequent occurrence of
resistant disease in T-ALL. Subsequently, patients were classified
according to their MRD level at time point 1 (TP1), taken at the end of
induction treatment (5 weeks), and at TP2 just before the start of
consolidation treatment (3 months). Patients were considered at low risk
if TP1 and TP2 were MRD negative and at high risk if MRD levels at TP1 and
TP2 were 10(-3) or higher; remaining patients were considered at
intermediate risk. The relative distribution of patients with T-ALL (n =
43) over the MRD-based risk groups differed significantly from that of
precursor B-ALL (n = 109). Twenty-three percent of patients with T-ALL and
46% of patients with precursor B-ALL were classified in the low-risk group
(P =.01) and had a 5-year relapse-free survival (RFS) rate of 98% or
greater. In contrast, 28% of patients with T-ALL were classified in the
MRD-based high-risk group compared to only 11% of patients with precursor
B-ALL (P =.02), and the RFS rates were 0% and 25%, respectively (P =.03).
Not only was the distribution of patients with T-ALL different over the
MRD-based risk groups, the prognostic value of MRD levels at TP1 and TP2
was higher in T-ALL (larger RFS gradient), and consistently higher RFS
rates were found for MRD-negative T-ALL patients at the first 5 follow-up
time points
Importance of allogeneic T-cells for disease control after stem cell transplantation for high-risk Langerhans cell histiocytosis
Reduced intensity conditioning followed by allogeneic SCT (RIC-SCT) has recently emerged as promising new salvage option for children suffering from Langerhans cell histiocytosis (LCH) with risk organ involvement and failure to conventional therapy. We report on the posttransplant course of female toddler with high-risk LCH, who achieved complete remission after RIC-SCT, despite a posttransplant chimerism constellation, in which only the T-cell subset proved to be of donor origin in the long-term. We therefore suggest that allogeneic T-cells have played a crucial role in controlling disease activity in this patient and may exert the major curative effect after RIC-SCT for LCH
High efficacy of the MACOP-B regimen in the treatment of adult Langerhans cell histiocytosis, a 20 year experience
BACKGROUND: Adult Langerhans cell histiocytosis (LCH) is an orphan disease. Chemotherapy is usually reserved to patients presenting with single system multifocal (SS-m) or multisystem (MS) disease but due to the lack of randomized studies no standard first line therapy has been defined yet. Pediatric regimens based on the vinblastine/prednisone backbone are not well tolerated in adults and probably less effective. We previously demonstrated high efficacy of the dose dense polichemotherapy regimen MACOP-B in 7 adult patients with SS-m or MS-LCH, in terms of high response rate and durable responses. Here we report an update of these data with the purpose of evaluating the long term efficacy of MACOP-B in adult LCH. METHODS: Clinical data of all adult LCH patients (n = 17) diagnosed and treated at our Institution during the past 20-year period were retrospectively reviewed. RESULTS: A total of 11 patients (6 with SS-m and 5 with MS-LCH) were treated with MACOP-B from 1995 to 2014. The overall response rate was confirmed to be 100 %, with a complete response of 73 % and a partial response rate of 27 %. Overall progression free survival was 64 %, and disease free survival after achievement of initial CR was 87 %. Overall survival rate was 82 % after 6.7 years of median follow-up. CONCLUSIONS: These data confirm high activity of MACOP-B in adult LCH, indicating that a substantial fraction of patients achieve long lasting responses and can be cured with this therapeutic approach
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