Outcome of relapsed infant acute lymphoblastic leukemia treated on the interfant-99 protocol

Driessen, Emma M. C.; Lorenzo, Paola De; Campbell, Myriam; Felice, Marı́a Sara; Ferster, Alina; Hann, Ian; Vora, Ajay; Hovi, Liisa; Escherich, Gabriele; Li, C. K.; Mann, Georg; Leblanc, Thierry; Locatelli, Franco; Biondi, Andrea; Rubnitz, Jeffrey E.; Schrappe, Martin; Silverman, Lewis B.; Starý, Jan; Suppiah, Ram; Szczepański, Tomasz; Valsecchi, Maria Grazia; Pieters, Rob

doi:10.1038/leu.2015.246

Cited by 50 publications

(49 citation statements)

References 15 publications

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“…Importantly, the toxicity was also similar in the neonatal cases compared to the older infants but, perhaps because of the biological characteristics of the neonatal cases (including the CD10‐negative immunophenotype and high white cell count) the relapse rate was extremely high: 19/26 cases (73%) where complete remission was achieved relapsed and this was significantly higher than in the older infants (Van der Linden et al , ). Although some children with relapsed infant ALL may achieve a second remission (Tomizawa et al , ; Driessen et al , ), the small numbers of surviving children make it difficult to assess the best approach to salvage therapy for neonatal ALL. Interestingly, in a study from the Japanese Infant Leukaemia Study Group, 3 of the 8 cases of neonatal ALL were reported to be alive in complete remission more than 3 years after diagnosis following haemopoietic stem cell transplantation (HSCT) in first ( n = 2) or second ( n = 1) remission, indicating that HSCT is feasible in this situation and can be curative (Ishii et al , ).…”

Section: Managementmentioning

confidence: 99%

Neonatal leukaemia

et al. 2018

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Neonatal leukaemia is defined as occurring within the first 28 days of life and most, if not all, cases are congenital. With the exception of Down syndrome-associated transient abnormal myelopoiesis, which is not considered here, neonatal leukaemias are rare. In two-thirds of patients the disease manifests as an acute myeloid leukaemia, frequently with monocytic/monoblastic characteristics. Most other cases are acute lymphoblastic leukaemia, particularly B lineage, but some are mixed phenotype or blastic plasmacytoid dendritic cell neoplasms. The most frequently observed cytogenetic/molecular abnormality is t(4;11)(q21.3;q23.3)/KMT2A-AFF1 followed by t(1;22)(p13.3;q13.1)/RBM15-MKL1 and t(8;16)(p11.2;p13.3)/KAT6A-CREBBP. Common clinical features include prominent hepatosplenomegaly and a high incidence of skin involvement, sometimes in the absence of bone marrow disease. A distinctive feature is the occurrence of spontaneous remission in some cases, particularly in association with t(8;16). In this review, we summarise current knowledge of the clinical, cytogenetic and molecular features of neonatal leukaemia and discuss clinical management of these cases.

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Section: Managementmentioning

confidence: 99%

Neonatal leukaemia

et al. 2018

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“…Depending on the protocol, indication to HSCT differs, and the advantage derived from transplantation performed in CR1 still remains debatable. For the European and Japanese cooperative groups, HSCT significantly increased OS and eventfree survival for high-risk patients transplanted in CR1 [16,19]. However, reports by other groups did not show advantages of using HSCT [7].…”

Section: Discussionmentioning

confidence: 96%

Unrelated Cord Blood Transplantation for Acute Leukemia Diagnosed in the First Year of Life: Outcomes and Risk Factor Analysis

Ruggeri

Volt

Locatelli

et al. 2017

Biology of Blood and Marrow Transplantation

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Infant acute leukemia still has a poor prognosis, and allogeneic hematopoietic stem cell transplantation is indicated in selected patients. Umbilical cord blood (UCB) is an attractive cell source for this population because of the low risk of chronic graft-versus-host disease (GVHD), the strong graft-versus-leukemia effect, and prompt donor availability. This retrospective, registry-based study reported UCB transplantation (UCBT) outcomes in 252 children with acute lymphoblastic leukemia (ALL; n = 157) or acute myelogenous leukemia (AML; n = 95) diagnosed before 1 year of age who received a single-unit UCBT after myeloablative conditioning between 1996 and 2012 in European Society for Blood and Marrow Transplantation centers. Median age at UCBT was 1.1 years, and median follow-up was 42 months. Most patients (57%) received a graft with 1 HLA disparity and were transplanted in first complete remission (CR; 55%). Cumulative incidence function (CIF) of day 100 acute GVHD (grades II to IV) was 40% ± 3% and of 4-year chronic GVHD was 13% ± 2%. CIF of 1-year transplant-related mortality was 23% ± 3% and of 4-year relapse was 27% ± 3%. Leukemia-free-survival (LFS) at 4 years was 50% ± 3%; it was 40% and 66% for those transplanted for ALL and AML, respectively (P = .001). LFS was better for patients transplanted in first CR, regardless of diagnosis. In multivariate model, diagnosis of ALL (P = .001), advanced disease status at UCBT (<.001), age at diagnosis younger than 3 months (P = .012), and date of transplant before 2004 were independently associated with worse LFS. UCBT is a suitable option for patients diagnosed with infant acute leukemia who achieve CR. In this cohort, patients with AML had better survival than those with ALL.

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“…Infant patients with R/R ALL have poor outcomes, and few studies have focused on this specific group. 223,224 Studies summarized previously for B-ALL and T-ALL include some infant patients, and those management strategies apply in this context.…”

Section: Management Of Infant Patients With Relapsed or Refractory Allmentioning

confidence: 99%

Pediatric Acute Lymphoblastic Leukemia, Version 2.2020, NCCN Clinical Practice Guidelines in Oncology

Brown

Inaba

Annesley

et al. 2020

Journal of the National Comprehensive Cancer Network

124

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Acute lymphoblastic leukemia (ALL) is the most common pediatric malignancy. Advancements in technology that enhance our understanding of the biology of the disease, risk-adapted therapy, and enhanced supportive care have contributed to improved survival rates. However, additional clinical management is needed to improve outcomes for patients classified as high risk at presentation (eg, T-ALL, infant ALL) and who experience relapse. The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for pediatric ALL provide recommendations on the workup, diagnostic evaluation, and treatment of the disease, including guidance on supportive care, hematopoietic stem cell transplantation, and pharmacogenomics. This portion of the NCCN Guidelines focuses on the frontline and relapsed/refractory management of pediatric ALL.

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Outcome of relapsed infant acute lymphoblastic leukemia treated on the interfant-99 protocol

Cited by 50 publications

References 15 publications

Neonatal leukaemia

Neonatal leukaemia

Unrelated Cord Blood Transplantation for Acute Leukemia Diagnosed in the First Year of Life: Outcomes and Risk Factor Analysis

Pediatric Acute Lymphoblastic Leukemia, Version 2.2020, NCCN Clinical Practice Guidelines in Oncology

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