Impact of the type of the BCR-ABL fusion transcript on the molecular response in pediatric patients with chronic myeloid leukemia

Suttorp, Meinolf; Thiede, Christian; Tauer, Josephine T.; Range, Ursula; Schlegelberger, Brigitte; Neuhoff, Nils von

doi:10.3324/haematol.2009.019224

Cited by 6 publications

(5 citation statements)

References 8 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…There seems to be a trend for inferior response rates in both adult and pediatric patients if they present with an e13a2 type of the BCR-ABL transcript compared with the e14a2 transcript type. 45,46 …”

Section: Treatment Responsementioning

confidence: 99%

See 1 more Smart Citation

Treatment of Pediatric Chronic Myeloid Leukemia in the Year 2010: Use of Tyrosine Kinase Inhibitors and Stem-Cell Transplantation

Suttorp

Millot²

2010

Hematology

View full text Add to dashboard Cite

Allogeneic hematopoietic stem cell transplantation (allo-SCT) remains the only proven cure for chronic myeloid leukemia (CML), a rare malignancy in childhood. With the excellent results induced by the tyrosine kinase inhibitor (TKI) imatinib in adults in the last decade, the appropriate management of children with CML has also changed radically, and only a minority are now transplanted as a front-line treatment. Data on pediatric experiences with imatinib in CML from controlled trials remain very limited, but this review of available data describes the role of imatinib in children with CML, addressing: 1) the starting dose; 2) pharmacokinetics in childhood; 3) possible adverse effects, with a focus on the still-growing skeleton; 4) early monitoring of treatment efficacy in an attempt to avoid failure; 5) the timing of allo-SCT in children; and 6) treatment of CML relapse after allo-SCT. Because the characteristics of CML in children seem to overlap extensively with what is described in adult internal medicine, most answers and pediatric algorithms are adapted from the treatment of CML in adults. Today in 2010, allo-SCT in children should be postponed until CML becomes refractory to imatinib. The approach for young patients with suboptimal responses is unclear because data on the efficacy and safety of second-generation TKIs in childhood are almost entirely missing. Other than being included in a formal trial on second-generation TKIs, allo-SCT for patients failing imatinib remains the first choice.

show abstract

Section: Treatment Responsementioning

confidence: 99%

“…However, the early response to IMA is a clinically relevant parameter for judging the aggressiveness of CML. 8,10,45,46 An established scoring system allows estimating of transplant-related mortality in pediatric patients pre-SCT.…”

Section: Arguments For and Against Allo-sct And Timing Of The Proceduresmentioning

confidence: 99%

Treatment of Pediatric Chronic Myeloid Leukemia in the Year 2010: Use of Tyrosine Kinase Inhibitors and Stem-Cell Transplantation

Suttorp

Millot²

2010

Hematology

View full text Add to dashboard Cite

show abstract

“…11 It has been shown that the presence of certain specific BCR-ABL1 transcripts determine clinical response. Suttorp et al 12 documented in pediatric patients with CML and e14a2 (b3a2) transcript have a faster response to Imatinib in comparison to those who present with e13a2 (b2a2) variant. 12 However, due to the rarity of patients with e1a2 transcript there is no documented association between response to imatinib which may explain the suboptimal response of the present case.…”

Section: Discussionmentioning

confidence: 99%

“…Suttorp et al 12 documented in pediatric patients with CML and e14a2 (b3a2) transcript have a faster response to Imatinib in comparison to those who present with e13a2 (b2a2) variant. 12 However, due to the rarity of patients with e1a2 transcript there is no documented association between response to imatinib which may explain the suboptimal response of the present case. On the other hand, prognostic classification systems used in adult patients lack any value in pediatric patients.…”

Section: Discussionmentioning

confidence: 99%

Variant p190 of the BCR-ABL1 gene in a pediatric patient with chronic myeloid leukemia

Angulo¹

2019

IJFCM

View full text Add to dashboard Cite

Chronic myeloid leukemia is a clonal disease characterized by the overproduction of cells of the myeloid lineage, this entity affects mainly adults, describing a low frequency in the pediatric population. The development of the disease is due to the formation of the BCR-ABL1 fusion gene, the 190 kDa product being an infrequent finding. We present the case of an 11-year-old patient whose disease started 2 months before diagnosis, characterized by weight loss. Physical examination revealed parenchymal pallor and splenomegaly with an analytical test showing leukocytosis with the presence of myeloid precursors, for which morphological, cytogenetic and molecular study of bone marrow was performed. It was established the diagnosis of CML in chronic phase with the unusual presence of the transcript p190. Therefore, initial treatment with imatinib was indicated, showing a suboptimal response, being replaced by dasatinib, once a major molecular response was documented, a transplant of hematopoietic stem cells from a related compatible donor was scheduled. Currently the patient is 17 years old and molecular tests show that he is free of the disease.

show abstract

“…In pediatric cohorts with CML the frequency of the two transcript types has been analyzed showing a slightly higher incidence of e14a2 than in adults. However, the prognostic relevance and significance of either transcript type for therapy or long-term survival have not been clearly defined due to the rarity of the disease [ 70 , 127 ].…”

Section: Prognosis and Predictionmentioning

confidence: 99%

Definition, Epidemiology, Pathophysiology, and Essential Criteria for Diagnosis of Pediatric Chronic Myeloid Leukemia

Suttorp

Millot

Sembill

et al. 2021

Cancers

View full text Add to dashboard Cite

Depending on the analytical tool applied, the hallmarks of chronic myeloid leukemia (CML) are the Philadelphia Chromosome and the resulting mRNA fusion transcript BCR-ABL1. With an incidence of 1 per 1 million of children this malignancy is very rare in the first 20 years of life. This article aims to; (i) define the disease based on the WHO nomenclature, the appropriate ICD 11 code and to unify the terminology, (ii) delineate features of epidemiology, etiology, and pathophysiology that are shared, but also differing between adult and pediatric patients with CML, iii) give a short summary on the diseases to be considered as a differential diagnosis of pediatric CML, (iv) to describe the morphological, histopathological and immunophenotypical findings of CML in pediatric patients, (v) illustrate rare but classical complications resulting from rheological problems observed at diagnosis, (vi) list essential and desirable diagnostic criteria, which hopefully in the future will help to unify the attempts when approaching this rare pediatric malignancy.

show abstract

Impact of the type of the BCR-ABL fusion transcript on the molecular response in pediatric patients with chronic myeloid leukemia

Cited by 6 publications

References 8 publications

Treatment of Pediatric Chronic Myeloid Leukemia in the Year 2010: Use of Tyrosine Kinase Inhibitors and Stem-Cell Transplantation

Treatment of Pediatric Chronic Myeloid Leukemia in the Year 2010: Use of Tyrosine Kinase Inhibitors and Stem-Cell Transplantation

Variant p190 of the BCR-ABL1 gene in a pediatric patient with chronic myeloid leukemia

Definition, Epidemiology, Pathophysiology, and Essential Criteria for Diagnosis of Pediatric Chronic Myeloid Leukemia

Contact Info

Product

Resources

About