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

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.

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.

 

 

Leukostasis in children and adolescents with chronic myeloid leukemia: Japanese pediatric leukemia/lymphoma study group (Kurosawa H et al. Pediatr Blood Cancer, October 2015)

Pediatr Blood Cancer. 2015 Oct 20. doi: 10.1002/pbc.25803. [Epub ahead of print]
Kurosawa H et al. 
 

Abstract:

 

Background:  

The details of leukostasis in children and adolescents with chronic myeloid leukemia (CML) are unknown. This study determined the characteristics of leukostasis in children and adolescents with CML.

PROCEDURE: 

A total of 256 cases from a retrospective study of patients with CML conducted by the Japanese Pediatric Leukemia/Lymphoma Study Group from 1996 to 2011 were analyzed, and of these, 238 cases were evaluated in this study.

RESULTS: 

Leukostasis was diagnosed in 23 patients (9.7%). The median leukocyte count and spleen size below the left costal margin in cases with leukostasis were significantly higher and larger when compared to those in cases without leukostasis (458.5 × 109 /l vs. 151.8 × 109 /l (P < 0.01), and 13 vs. 5 cm (P < 0.01), respectively). Leukostasis occurred with ocular symptoms in 14 cases, priapism in four cases, and dyspnea, syncope, headache, knee pain, difficulty hearing, and aseptic necrosis of the femoral head in one case each. One case had two leukostasis symptoms simultaneously. Three cases were diagnosed before imatinib became available. Five cases received special treatment, and in the remaining 15 cases, all of these symptoms resolved after treatment with imatinib.

CONCLUSIONS: 

This retrospective study represents the largest series of children and adolescents in which leukostasis of CML has been reported. Our data provide useful insight into the characteristics of leukostasis in recent cases of children and adolescents with CML.

 

© 2015 Wiley Periodicals, Inc.

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.

Atypical chronic myeloid leukemia in two pediatric patients (Freedman JL et al. Pediatr Blood Cancer, August 2015)

Pediatr Blood Cancer. 2015 Aug 14. doi: 10.1002/pbc.25694. [Epub ahead of print]

Freedman JL et al.

 

Abstract

Atypical chronic myeloid leukemia, BCR-ABL1-negative, (aCML) is a rare myeloid neoplasm. Recent adult data suggest the leukemic cells in a subset of patients are dependent on JAK/STAT signaling and harbor CSF3R-activating mutations. We hypothesized that, similar to adult patients, the presence of CSF3R-activating mutations would be clinically relevant in pediatric myeloid neoplasms as patients would be sensitive to the JAK inhibitor, ruxolitinib. We report two cases of morphologically similar pediatric aCML, BCR-ABL1-negative based on WHO 2008 criteria. One patient had CSF3R-activating mutation (T618I) and demonstrated a robust response to ruxolitinib, which was used to bridge to a successful stem cell transplant. The other patient did not have a CSF3R-activating mutation and succumbed to refractory disease <6 months from diagnosis. This report documents CSF3R-T618I in pediatric aCML and demonstrates the efficacy of ruxolitinib in a pediatric malignancy. As the third documented case successfully treating aCML with ruxolitinib, this case highlights the importance of prompt CSF3R sequencing analysis for myeloproliferative and myelodysplastic/myeloproliferative neoplasms. Pediatr Blood Cancer © 2015 Wiley Periodicals, Inc.

© 2015 Wiley Periodicals, Inc.

  1. 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)
  2. 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)
  3. Chronic myloid leukemia in children: clinical findings, management, and unanswered questions (Hijija N et al. Pediatr Clin North Am, February, 2015)
  4. Growth deceleration in children treated with imatinib for chronic myeloid leukaemia (Millot F et al. Eur J Cancer, Oct. 2014)