Translate page

English English
en Englishbg Bulgarianzh-CN Chinese (Simplified)hr Croatiancs Czechda Danishnl Dutchfi Finnishfr Frenchde Germanel Greekiw Hebrewhi Hindiid Indonesianit Italianja Japaneseko Koreanmk Macedonianpl Polishpt Portuguesero Romanianru Russiansr Serbiansk Slovaksl Slovenianes Spanishsv Swedishth Thaitr Turkishvi Vietnamese

Outcomes following bone marrow transplantation in children with accelerated phase or blast crisis chronic myelogenous leukemia in the era of tyrosine kinase inhibitors (Shulman DS et al. J Pediatr Hematol, July 2016)

J Pediatr Hematol, July 2016 (epub ahead of print)
Shulman DS et al


Abstract

The management of chronic myelogenous leukemia (CML) in children changed dramatically with the introduction of tyrosine kinase inhibitors (TKIs). Unfortunately, outcomes for patients presenting in an advanced stage-accelerated phase or blast crisis CML-continues to be poor, requiring chemotherapy and allogeneic hematopoietic stem cell transplant (HSCT) to attempt cure. Integration of TKIs in the therapy of advanced CML is still an area of active investigation. There are little published data on TKI use in children with advanced stage CML. We performed a retrospective review of all children treated at our institution between January 1, 2010 and June 30, 2013, and identified 5 children, age 12 to 18 years, with advanced stage CML. All patients were treated with a TKI before HSCT and TKIs were restarted post-HSCT in 4/5 with a goal of continuing until 2 years posttransplant. At time of HSCT all were in a morphologic and cytogenetic remission; 1 patient had also achieved molecular remission. All patients are alive and in molecular remission at an average of 38 months (range, 14 to 51 mo) following transplant. Our experience indicates that TKIs are safe and well tolerated in children both pretransplant and posttransplant and may improve outcomes in this aggressive disease. 

Recognizing endocrinopathies associated with tyrosine kinase inhibitor therapy in children with chronic myelogenous leukemia (Samis J et al. Pediatr Blood Cancer, April 2016)

Pediatr Blood Cancer, April 2016 (epub ahead of print)
Samis J et al.

 

Abstract

Side effects of tyrosine kinase inhibitor (TKI) treatment vary in children and adults with chronic myelogenous leukemia (CML). As children have a much longer life expectancy than adults, TKI therapy may continue for decades and with long-term consequences that differ from adults. Children may develop endocrinopathies related to "off-target" effects of TKIs, such as delayed growth, changes in bone metabolism, thyroid abnormalities, and effects on puberty and fertility. These endocrinopathies present additional challenges for pediatric patients with CML. This review critically evaluates the literature on long-term endocrine side effects of TKIs in the pediatric CML population and provides suggested recommendations.

© 2016 Wiley Periodicals, Inc. 

 

Factors associated with improved outcomes after second allogeneic hematopoietic cell transplantation for relapsed pediatric leukemia (Manon NN et al, Ann Hematol, January 2016

Ann Hematol, January 2016 (Epub ahead of print)
Menon NN et al.

  

Abstract

A second allogeneic (allo) hematopoietic cell transplant (HCT) is an important therapeutic consideration for patients relapsing after their first. We conducted a retrospective review of 41 pediatric patients with leukemia that underwent a second allo-HCT at our institution. Overall, 53.7 and 43.9 % of patients were alive and disease-free at 1 and 5 years, respectively, after the second allo-HCT. The factors affecting outcome by both univariate and multivariate analysis were interval between transplants and the use of a myeloablative conditioning (MAC) regimen prior to second transplant. Outcomes were inferior in patients who received their second transplant <6 months from their first HCT when compared to patients in whom the interval between HCTs was 6-12 or more than 12 months. Interval between HCTs was also significant when each type of leukemia (acute lymphoblastic leukemia (ALL) n = 21, acute myelogenous leukemia (AML) n = 11, and chronic myelogenous leukemia (CML) n = 7) was analyzed separately. In univariate analysis, use of the same donor and use of a matched sibling donor resulted in significant improved outcome. There was not a significant association between disease-free survival (DFS) and age, remission status, use of total body irradiation (TBI) before second HCT, or type of leukemia. Second allogeneic HCT can be a curative therapeutic option for leukemia patients relapsing after their first transplant. As more targeted therapies have become available, patients that relapse after first HCT are more likely to achieve remission. Therefore, it is anticipated that there will be more candidates for second HCT with improved performance and remission status, ultimately leading to a better outcome with the second HCT.

 

 

Can chronic myleoid leukaemia in children and adolescents be successfully treated with haematopoietic stem cell transplant? A single center experience (Giona F et al. Br J Haematol, February 2016)

Br J Haematol, February 2016 (epub ahead of print)
Giona F et al.

 

Abstract

We analysed the long-term outcome of 35 children and adolescents (<20 years at diagnosis) with chronic myeloid leukaemia (CML) in chronic phase: 20 patients had received interferon-alpha and/or tyrosine kinase inhibitors (TKIs), and 15 underwent a haematopoietic stem cell transplant. The 10-year survival probabilities were similar in transplanted and non-transplanted patients (73·3% vs. 72·1%, respectively), whereas the survival probability was significantly lower in patients diagnosed before 1999 compared to those diagnosed afterwards (62·1% vs. 100%, P = 0·0384). The availability of TKIs and the standardized molecular monitoring have significantly improved treatment, management and outcome in children and adolescents with CML.

 

© 2016 John Wiley & Sons Ltd.

Pediatric chronic myeloid leukemia is a unique disease that requires a different approach (Hijiya N et al. Blood, October 2015)

Blood, 2015 Oct 28 (Epub ahead of print)
Hijiya N. et al.

 

Abstract

Chronic myelogenous leukemia (CML) in children is relatively rare. Due to lack of robust clinical study evidence, management of CML in children is not standardized and often follows guidelines developed for adults. Children and young adults tend to have a more aggressive clinical presentation than older adults, and prognostic scores for adult CML do not apply to children. CML in children has been considered to have the same biology as in adults, but recent data indicate some genetic differences exist in pediatric and adult CML. As children with CML may receive tyrosine kinase inhibitor (TKI) therapy for many decades, and are exposed to TKIs during a period of active growth, morbidities in children with CML may be distinct from those in adults and require careful monitoring. Aggressive strategies, such as eradication of CML stem cells with limited duration and intensive regimens of chemotherapy and TKIs, may be more advantageous in children as a way to avoid life-long exposure to TKIs and their associated adverse effects. Blood and marrow transplantation (BMT) in pediatric CML is currently indicated only for recurrent progressive disease, and the acute and long-term toxicities of this option should be carefully evaluated against the complications associated with lifelong use of TKIs.

Copyright © 2015 American Society of Hematology.

  1. Leukostasis in children and adolescents with chronic myeloid leukemia: Japanese pediatric leukemia/lymphoma study group (Kurosawa H et al. Pediatr Blood Cancer, October 2015)
  2. Atypical chronic myeloid leukemia in two pediatric patients (Freedman JL et al. Pediatr Blood Cancer, August 2015)
  3. Long-term results of high-dose imatinib in children and adolescents with CML in chronic phase: The Italian experience (Giona F et al. Br J Haematol, Apr 2015)
  4. Can prognostic scoring systems for chronic myeloid leukemia as established in adults be applied to pediatric patients? (Gurrea Salas D et al. Ann Hematol, Apr 2015)