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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.

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.

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)

Ann Hematol. 2015 Apr 19. [Epub ahead of print]

Gurrea Salas D et al 


Abstract

In contrast to adult medicine, specific scoring systems predicting the treatment response for an individual pediatric patient (pt) with chronic myeloid leukemia (CML) have not yet been defined. We evaluated to what extend prognostic scores as described for adults (e.g., Sokal, Hasford, EUTOS score) resulted in comparable risk group categorizations in a pediatric cohort. Parameters for score calculation were extracted from a data set of 90 patients enrolled into trial CML-PAED-II and treated by a standard dose of imatinib. At month 3 and at month 6, treatment response was analyzed based on the transcript ratio BCR-ABL1/ABL1. By the EUTOS, Hasford, and Sokal scores 81, 59, and 62 % of the patients were categorized as low risk, respectively; 19, 14, and 16 % of the patients as high risk, respectively; and by Hasford and Sokal scores 27 and 22 % of the patients, respectively, as intermediate risk. Twenty-seven out of 72 patients analyzable (38 %) exhibited a transcript ratio >10 % at month 3. We show that only the EUTOS score, but not the Sokal and Hasford score, correlates with this early outcome (p = 0.008). Analyzing the EUTOS score separately, we can demonstrate that lowering the cutoff from 87 to 48 points for categorization in low- and high-risk individuals increases the odds ratio from 2.4 (95 % CI 0.6 to 10.4) to 3.6 (95 % CI 1.3 to 10.9). Data are provided on the distribution of risk categories and resulting discrepancies when adult scores are applied on children and adolescents with CML at diagnosis. A larger number of patients and longer follow-up are still needed to develop a prognostic score specifically adapted to the pediatric and adolescent age cohorts.

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)

 

Br J Haematol. 2015 Apr 20. doi: 10.1111/bjh.13453. [Epub ahead of print]
Giona F et al.


Abstract

Imatinib mesylate (IM) is used for the management of childhood chronic myeloid leukaemia (CML). The most effective dosage of IM and its long-term efficacy in children are not well defined. The purpose of this multicentre study is to report on the long-term results of high-dose IM (340 mg/m2 /d) in CML patients in chronic phase (CP-CML) aged <18 years at diagnosis. A total of 47 CP-CML patients with a median age at diagnosis of 11 years 9 months were enrolled in nine Italian centres. Complete cytogenetic response was achieved in 91·5% of the evaluable patients at a median time of 6 months. BCR-ABL1 International Scale ≤ 0·1% (major molecular response; MMR) and ≤0·01% (molecular response; MR) at 12 months were 66·6% and 33%, respectively. During follow-up, MMR and MR were achieved in 78·6% and 61% of children, respectively. IM was safely discontinued in 3 long-term treated children with a durable MR. Twelve patients (eight cytogenetic/molecular responders) underwent stem cell transplantation. The progression-free survival probabilities at 96 months for responding patients who continued IM and for those transplanted were 60% and 50%, respectively. After a median follow-up of 52 months (range 3-146), all patients are alive. High-dose IM is a long-term effective therapy in children and adolescents with CP-CML.

Chronic myloid leukemia in children: clinical findings, management, and unanswered questions (Hijija N et al. Pediatr Clin North Am, February, 2015)

Pediatr Clin North Am. 2015 Feb;62(1):107-19. doi: 10.1016/j.pcl.2014.09.008.

Hijiya N, Millot F, Suttorp M.


Abstract

Chronic myelogenous leukemia (CML) is a rare disease in children. There is little evidence of biological differences between CML in children and adults, although host factors are different. Children develop distinct morbidities related to the off-target effects of tyrosine kinase inhibitors. The goal of treatment in children should be cure rather than suppression of disease, which can be the treatment goal for many older adults. This article reviews data from the literature on the treatment of CML, discusses the issues that are unique to CML in children, and recommends management that takes these issues into consideration.


Copyright © 2015 Elsevier Inc. All rights reserved.

  1. Growth deceleration in children treated with imatinib for chronic myeloid leukaemia (Millot F et al. Eur J Cancer, Oct. 2014)
  2. Differences among young adults, adults and elderly chronic myeloid leukemia patients (Castagnetti F et al. Ann. Oncol, Oct. 2014)
  3. Managing children with CML: Pediatric CML Guideline if IBFM (Fuente et al. Br J Haematology, July 2014)
  4. Imatinib cessation in children and adolescents with chronic myeloid leukemia in chronic phase (Millot et al. Pediatr Blood Cancer, Feb. 2014)