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Ching-Hon Pui

Publications and source records attributed to Ching-Hon Pui.

At least 19 recordsLinked to original sources

The 21st International ``Ponte di Legno'' Childhood Acute Lymphoblastic Leukemia Workshop Report: Progress and Emerging Opportunities.

The 21st Ponte di Legno Working Group meeting convened in Orlando, USA, on December 4-5, 2025, bringing together leading childhood acute lymphoblastic leukemia (ALL) investigators from major global consortia. In response to the transformative advances in childhood ALL treatment, particularly the integration of immunotherapy into frontline therapy, the group revisited and updated its mission statement. The revised mission emphasizes collaborative studies on rare leukemia subsets, harmonized toxicity reporting, particularly for immunotherapy-related toxicities, and unrestricted worldwide collaboration. In addition, sharing data and strategies for integrating novel agents will be integral to optimizing future trial design. Key scientific topics included rare genetic subgroups, T-cell ALL genomics, treatment-related toxicity benchmarking, and central nervous system (CNS) disease management challenges. A major focus was immunotherapy integration into frontline therapy, particularly blinatumomab as an emerging standard of care and inotuzumab ozogamicin as an investigational agent, and their potential to enable chemotherapy de-escalation. Additional discussions addressed immunotherapy-specific toxicities. The integration of immunotherapy into frontline ALL therapy represents a paradigm shift with potential to improve outcomes while reducing treatment burden. However, careful attention to CNS disease control, emerging toxicities, and preservation of the remarkable achievements in childhood ALL therapy remains essential as the field advances.

Blinatumomab↗

Prognostic factors and outcome of recurrence in childhood acute myeloid leukemia.

BACKGROUND: Outcome after recurrence of childhood acute myeloid leukemia (AML) is poor. We performed this study to identify prognostic factors for recurrence and for survival after recurrence of AML. METHODS: The clinical characteristics, biological features, treatment modalities, and outcomes of children with de novo AML who were enrolled on 3 consecutive clinical protocols from 1987 to 2002 at St. Jude Children's Research Hospital were studied. Regression modeling was used to identify prognostic factors for recurrence and for survival after recurrence. RESULTS: The outcome after recurrence was poor, with a 5-year survival estimate of only 23.3% +/- 5.7%. Multivariable analysis indicated that male sex (P = .005), autologous stem cell transplant before recurrence (P = .097), each additional month from diagnosis to recurrence (P = .041), and stem cell transplant after recurrence (P < .001) were associated with a better survival after recurrence, whereas M5 or M7 morphology (P = .001) were significantly predictive of a lower survival estimate after recurrence. CONCLUSIONS: Survival after recurrence was poor in children with AML. Novel therapies are urgently needed to prevent or to treat recurring AML.

Acute Disease↗

Effect of race on outcome of white and black children with acute myeloid leukemia: the St. Jude experience.

BACKGROUND: The association between race and outcome of treatment for childhood acute myeloid leukemia (AML) has not been adequately studied. PROCEDURE: We compared the clinical characteristics, biological features, and outcomes between white and black children with AML who were treated on five consecutive clinical protocols (1980-2002) at St. Jude Children's Research Hospital. We used proportional hazards modeling to investigate the relation between race and outcome. RESULTS: We observed no statistically significant differences between the 229 white and 58 black patients in clinical characteristics, FAB subtype, cytogenetic features, or outcome. There were no significant differences in event-free survival (EFS) or overall survival (OS) between the two race groups in individual clinical trials or in all studies combined. For the study group as a whole, the 5-year survival estimate was 39.2% +/- 3.6% for white patients and 33.8% +/- 6.5% for black patients. However, on our most recent trial (AML-97), there was a trend towards inferior outcome among black patients: the 5-year survival estimates were 55.6% +/- 12.3% and 27.3% +/- 13.5% for whites and blacks, respectively. CONCLUSIONS: Although we detected no differences in treatment outcome between white and black children with AML over the entire study period, black children appear to have worse outcomes than white children during more recent studies. Improved treatment is needed for all children with AML.

Adolescent↗

Comparison of treatment outcomes of childhood Hodgkin lymphoma in two US centers and a center in Recife, Brazil.

BACKGROUND: Pediatric Hodgkin lymphoma (HL) has a cure rate of more than 80% in high-income countries (HIC). However, more than 80% of the world's children live in low-income countries (LIC), where the cure rate is often much lower. PROCEDURE: We compared the outcome of HL of 371 patients treated at two pediatric oncology centers in the US to that of 62 patients treated at one center in Recife, Brazil (IMIP) to determine whether the same treatment strategy should be used in both high-income and LIC. The logrank test was used to compare event-free and overall survival. RESULTS: The percentages of patients with unfavorable disease at each center were similar (P = 0.72). Patients with favorable disease at IMIP had estimated 5-year survival rates comparable to those of the US centers (100% and 99%, respectively). Among patients with unfavorable disease, those treated at IMIP had a 5-year event-free survival (EFS) rate of 60%, compared to 78% at the US centers; (P = 0.08). The 5-year survival estimate after relapse was 25% at IMIP versus 61% at the US centers (P = 0.08). The 5-year overall survival for patients with unfavorable disease was 72% at IMIP versus 90% at the US centers (P = 0.01). CONCLUSIONS: Intensive frontline therapy should be considered for patients with unfavorable HL in LIC where the relapse rate is high and the salvage rate is low, provided that supportive care is adequate.

Adolescent↗

Implementation of a data management program in a pediatric cancer unit in a low income country.

INTRODUCTION: Pediatric cancer units in low-income countries lack data on which to base quality improvement initiatives. We implemented a data management program in the oncology unit of the children's hospital of Tegucigalpa, Honduras, and then we assessed training and supervision of data managers, data accuracy, and completeness as well as obstacles encountered. METHODS: Training included 2 days of off-site hands-on instruction in the use of an online database, daily on-site supervision by physicians, periodic online meetings for education and problem-solving, and continuous e-mail support. RESULTS: Of the 652 patients diagnosed with acute leukemia between July 1995 and June 2005, 150 (23%) had not yet been registered in the database at the time of audit and 65 (10%) had missing medical records. The remaining 437 charts (67%) were reviewed by an external auditor and compared to the data entered previously by the two trained data managers. Protocol information was incomplete in 30% of cases, and the cause of death was inaccurate in 18%. All other data were 99% accurate and 93%-100% complete. Obstacles included a limited medical records system, poor organization of the charts, missing records, inconsistently documented protocol information, data managers who lack a medical background, and slow or unreliable internet connections. CONCLUSION: Data managers can be trained to effectively collect basic pediatric oncology data in a low-income country. Addressing inadequacies in the medical record system while providing specific training in protocol-based care and determination of cause of death for both physicians and data managers will improve data quality.

Clinical Protocols↗

Improving outcomes for children with cancer in low-income countries in Latin America: a report on the recent meetings of the Monza International School of Pediatric Hematology/Oncology (MISPHO)-Part I.

The difference in survival for children diagnosed with cancer between high- and low-income countries (LIC) continues to widen as curative therapies are developed in the former but not implemented in the latter. In 1996, the Monza International School of Pediatric Hematology/Oncology (MISPHO) was founded in an attempt to narrow this survival gap. During its sixth and seventh meetings, members recognized the problem of lack of affordability of essential drugs to treat childhood cancer in many LIC, and initiated an advocacy program. In 1998, MISPHO spawned a collaboration of Central American pediatric oncology centers: the Asociación de Hemato-Oncología Pediátrica Centroamericana (AHOPCA). AHOPCA members reported preliminary findings from several of the 10 cooperative protocols that are currently in progress. In 2003, a second regional collaborative group was formed that includes seven centers in South America. Twinning programs between MISPHO centers and centers in high-income countries (HIC) have proven invaluable to harness the resources of these centers to improve pediatric oncology care in LIC. MISPHO educational efforts include oncology nursing, supportive care, cancer-specific updates, epidemiology, and clinical research methods. Educational efforts are facilitated by educational content and online conferencing via www.cure4kids.org. Identifying preventable causes of abandonment of therapy and documenting the nutritional status of patients treated at MISPHO centers are areas of active research.

Adolescent↗

Secondary chromosomal abnormalities predict outcome in pediatric and adult high-stage Burkitt lymphoma.

BACKGROUND: Karyotypic abnormalities in sporadic Burkitt lymphoma (BL) have been described extensively. However, to the authors' knowledge, very limited studies have focused on the secondary chromosomal abnormalities in pediatric BL as compared with those of adult BL and on their prognostic impact. METHODS: A retrospective analysis was performed in all pediatric and adult patients at 2 institutions, with a morphologic diagnosis of BL, pretherapy tumor karyotype available, and t(8;14), t(8;22), or t(2;8) present. RESULTS: There were 33 children and 37 adults. The majority of the patients (95%) had Stage III/IV disease. There were no statistically significant differences noted in karyotype complexity and the nature of the chromosomal abnormalities between these 2 groups. Abnormalities of chromosomes 13 (13q) and 22 (22q) had a negative impact on prognosis in children. In adults, abnormalities of chromosome 17 appeared to have a negative impact. CONCLUSIONS: The current findings suggest that karyotypic information can be used for refining risk stratification in patients with BL.

Adolescent↗

Body mass index does not influence pharmacokinetics or outcome of treatment in children with acute lymphoblastic leukemia.

There is conflicting information about the influence of body mass index (BMI) on the pharmacokinetics, toxicity, and outcome of chemotherapy. We compared pharmacokinetics, outcome, and toxicity data across 4 BMI groups (underweight, BMI < or = 10th percentile; normal; at risk of overweight, BMI > or = 85th and < 95th percentile; overweight, BMI > or = 95th percentile) in 621 children with acute lymphoblastic leukemia (ALL) treated on 4 consecutive St Jude Total Therapy studies. Chemotherapy doses were not adjusted to ideal BMI. Estimates of overall survival (86.1% +/- 3.4%, 86.0% +/- 1.7%, 85.9% +/- 4.3%, and 78.2% +/- 5.5%, respectively; P = .533), event-free survival (76.2% +/- 4.2%, 78.7% +/- 2.1%, 73.4% +/- 5.5%, and 72.7% +/- 5.9%, respectively; P = .722), and cumulative incidence of relapse (16.0% +/- 3.7%, 14.4% +/- 1.8%, 20.6% +/- 5.1%, and 16.7% +/- 5.1%, respectively; P = .862) did not differ across the 4 groups. In addition, the intracellular levels of thioguanine nucleotides and methotrexate polyglutamates did not differ between the 4 BMI groups (P = .73 and P = .74, respectively). The 4 groups also did not differ in the overall incidence of grade 3 or 4 toxicity during the induction or postinduction periods. Further, the systemic clearance of methotrexate, teniposide, etoposide, and cytarabine did not differ with BMI (P > .3). We conclude that BMI does not affect the outcome or toxicity of chemotherapy in this patient population with ALL.

Adolescent↗

Prognostic significance of CD20 expression in childhood B-cell precursor acute lymphoblastic leukemia.

CD20 expression is associated with inferior survival in adults with acute lymphoblastic leukemia (ALL). We analyzed the prognostic impact of CD20 expression in 353 children with B-cell precursor ALL treated in 3 consecutive St Jude Total Therapy studies. CD20 expression (> 20%) was found in 169 patients (48%) and was more frequent in patients between 1 and 10 years of age than in those younger than 1 or older than 10 years (P = .001). None of 14 patients with MLL-AF4 expressed CD20. There was no association between CD20 expression and E2A-PBX, TEL-AML1, ploidy, white blood cell count at diagnosis, or sex. In contrast to the experience in adult ALL, our patients with CD20 expression tended to have a better treatment outcome than those without the expression: 5-year event-free survival 84% +/- 2.9% versus 78% +/- 3.1% (P = .08). These data suggest that CD20 expression is not associated with inferior outcome in pediatric patients treated with contemporary regimens.

Age Factors↗

A subtle t(3;8) results in plausible juxtaposition of MYC and BCL6 in a child with Burkitt lymphoma/leukemia and ataxia-telangiectasia.

Translocations involving 3q27 that affect the BCL6 gene are common and specific chromosomal abnormalities in B-cell precursor non-Hodgkin lymphoma (mainly diffuse large-cell and follicular lymphoma), but they have not been reported in Burkitt lymphoma. Here, we describe a case in which a BCL6 rearrangement and additional complex cytogenetic abnormalities occurred in a child with Burkitt lymphoma/leukemia and ataxia-telangiectasia. Although cytogenetic analysis of the bone marrow revealed clonal abnormalities of chromosome arms 8q and 14p and other subclonal abnormalities, the t(8;14) or its variants typically associated with Burkitt lymphoma were not observed. Fluorescence in situ hybridization with locus-specific probes and multicolor spectral karyotyping demonstrated a complex pattern of chromosomal rearrangements leading to a subtle t(3;8)(q27;q24.1) that rearranged BCL6 and placed it adjacent to MYC. We speculate that this genetic lesion occurred as a result of chromosomal instability due to the underlying disease.

Ataxia Telangiectasia↗

Epigenetic regulation of human gamma-glutamyl hydrolase activity in acute lymphoblastic leukemia cells.

Gamma-glutamyl hydrolase (GGH) catalyzes degradation of the active polyglutamates of natural folates and the antifolate methotrexate (MTX). We found that GGH activity is directly related to GGH messenger RNA expression in acute lymphoblastic leukemia (ALL) cells of patients with a wild-type germline GGH genotype. We identified two CpG islands (CpG1 and CpG2) in the region extending from the GGH promoter through the first exon and into intron 1 and showed that methylation of both CpG islands in the GGH promoter (seen in leukemia cells from approximately 15% of patients with nonhyperdiploid B-lineage ALL) is associated with significantly reduced GGH mRNA expression and catalytic activity and with significantly higher accumulation of MTX polyglutamates (MTXPG(4-7)) in ALL cells. Furthermore, methylation of CpG1 was leukemia-cell specific and had a pronounced effect on GGH expression, whereas methylation of CpG2 was common in leukemia cells and normal leukocytes but did not significantly alter GGH expression. These findings indicate that GGH activity in human leukemia cells is regulated by epigenetic changes, in addition to previously recognized genetic polymorphisms and karyotypic abnormalities, which collectively determine interindividual differences in GGH activity and influence MTXPG accumulation in leukemia cells.

Adolescent↗

Impact of age on outcome of pediatric acute myeloid leukemia: a report from 2 institutions.

BACKGROUND: The prognostic significance of age among pediatric patients with acute myeloid leukemia (AML) was investigated. METHODS: The authors reviewed the outcome of 424 patients who were <or=21 years of age at the time of diagnosis of AML (excluding acute promyelocytic leukemia) between 1983 and 2002 at St. Jude Children's Research Hospital (n=288) or the M. D. Anderson Cancer Center (n=136). Two treatment eras (1983-1989 and 1990-2002) were examined because of the greater intensity of treatment during the recent era. RESULTS: After controlling for the effects of cytogenetics, white blood cell (WBC) count, French-American-British (FAB) subtype, and treatment era, they observed that age and treatment era interacted significantly in relation to event-free survival (EFS) (P=.033). Patients 10 years of age or older were at greater risk of an adverse event than younger patients in the recent era (hazard ratio=1.8; 95% confidence interval [CI]: 1.3-2.6; P=.005) but not in the early era. The rate of adverse events (death or recurrence) increased significantly with each year of age in the recent era (4.3%/year; 95% CI: 1.9-6.8%; P=.001) but not in the early era. The rate of death increased significantly with each year of age in both eras (4.4%/year; 95% CI: 2.3-6.5%; P<.001). EFS and survival showed no association with study site, and the effects of age were similar at the 2 sites. CONCLUSIONS: These results suggest that age is an independent prognostic factor in childhood AML and that children younger than 10 years benefit more than older children from newer intensive therapies.

Acute Disease↗

Expression of the outcome predictor in acute leukemia 1 (OPAL1) gene is not an independent prognostic factor in patients treated according to COALL or St Jude protocols.

New prognostic factors may result in better risk classification and improved treatment of children with acute lymphoblastic leukemia (ALL). Recently, high expression of a gene named OPAL1 (outcome predictor in acute leukemia) was reported to be associated with favorable prognosis in ALL. Therefore, we investigated whether OPAL1 expression was of prognostic importance in 2 independent cohorts of children with ALL treated on Cooperative Study Group for Childhood Acute Lymphoblastic Leukemia (COALL)-92/97 (n = 180) and St Jude Total 13 protocols (n = 257). We observed a consistently higher (2.8-fold) expression of OPAL1 in TEL-AML1-positive ALL compared with TEL-AML1-negative ALL in both cohorts, but higher OPAL1 expression was not consistently associated with other favorable prognostic indicators such as age and white blood cell count, or ALL genetic subtype. Lower OPAL1 expression was also not associated with increased in vitro drug resistance. Multivariate analyses including known risk factors showed that OPAL1 expression was not independently related to prognosis in either the COALL or St Jude cohorts. In conclusion, OPAL1 expression may not be an independent prognostic feature in childhood ALL, and its previously reported prognostic impact appears to be treatment dependent.

Antineoplastic Combined Chemotherapy Protocols↗

Computer-aided detection of therapy-induced leukoencephalopathy in pediatric acute lymphoblastic leukemia patients treated with intravenous high-dose methotrexate.

The purpose of this study was to use objective quantitative magnetic resonance imaging (MRI) methods to develop a computer-aided detection (CAD) tool to differentiate white matter (WM) hyperintensities into either leukoencephalopathy (LE) induced by chemotherapy or normal maturational processes in children treated for acute lymphoblastic leukemia without irradiation. A combined MRI set consisting of T1-weighted, T2-weighted, proton-density-weighted and fluid-attenuated inversion recovery images and WM, gray matter and cerebrospinal fluid proportional volume maps from a spatially normalized atlas were analyzed with a neural network segmentation based on a Kohonen self-organizing map (SOM). Segmented maps were manually classified to identify the most hyperintense WM region and the normal-appearing genu region. Signal intensity differences normalized to the genu within each examination were generated for four time points in 228 children. A second Kohonen SOM was trained on the first examination data and divided the WM into normal-appearing or LE groups. Reviewing labels from the CAD tool revealed a consistency measure of 89.8% (167 of 186) within patients. The overall agreement between the CAD tool and the consensus reading of two trained observers was 84.1% (535 of 636), with 84.2% (170 of 202) agreement in the training set and 84.1% (365 of 434) agreement in the testing set. These results suggest that subtle therapy-induced LE can be objectively and reproducibly detected in children treated for cancer using this CAD approach based on relative differences in quantitative signal intensity measures normalized within each examination.

Adolescent↗

Genes contributing to minimal residual disease in childhood acute lymphoblastic leukemia: prognostic significance of CASP8AP2.

In childhood acute lymphoblastic leukemia (ALL), early response to treatment is a powerful prognostic indicator. To identify genes associated with this response, we analyzed gene expression of diagnostic lymphoblasts from 189 children with ALL and compared the findings with minimal residual disease (MRD) levels on days 19 and 46 of remission induction treatment. After excluding genes associated with genetic subgroups, we identified 17 genes that were significantly associated with MRD. The caspase 8-associated protein 2 (CASP8AP2) gene was studied further because of its reported role in apoptosis and glucocorticoid signaling. In a separate cohort of 99 patients not included in the comparison of gene expression profiles and MRD, low levels of CASP8AP2 expression predicted a lower event-free survival (P = .02) and a higher rate of leukemia relapse (P = .01) and were an independent predictor of outcome. High levels of CASP8AP2 expression were associated with a greater propensity of leukemic lymphoblasts to undergo apoptosis. We conclude that measurement of CASP8AP2 expression at diagnosis offers a means to identify patients whose leukemic cells are highly susceptible to chemotherapy. Therefore, this gene is a strong candidate for inclusion in gene expression arrays specifically designed for leukemia diagnosis.

Adolescent↗

White race as a risk factor for hypothyroidism after treatment for pediatric Hodgkin's lymphoma.

PURPOSE: Hypothyroidism frequently occurs after treatment for pediatric Hodgkin's lymphoma, but race has not been investigated as a risk factor for this delayed toxicity. The aim of this study was to determine whether race is an independent risk factor for hypothyroidism in survivors of pediatric Hodgkin's lymphoma. PATIENTS AND METHODS: To identify differences between black and white patients in the development of hypothyroidism after treatment for Hodgkin's lymphoma, we conducted a retrospective study of consecutively treated pediatric patients with newly diagnosed Hodgkin's lymphoma treated at St Jude Children's Research Hospital (Memphis, TN) from January 1980 through December 2002. Clinical or biochemical hypothyroidism was defined by an above normal thyroxine-stimulating hormone concentration or by the need for thyroid hormone replacement therapy. RESULTS: The 461 patients (388 white patients, 73 black patients) where followed for a median of 11.3 years (range, 1.8 to 24.9 years). Hypothyroidism developed in 196 (43%) of 461 patients after a median of 2.9 years (range, 0.7 to 11.3 years) after diagnosis of Hodgkin's lymphoma. Hypothyroidism developed in 47% of white patients but in only 21% of black patients (hazard ratio = 2.7; 95% CI, 1.6 to 4.6). After adjusting for other risk factors for hypothyroidism (thyroid radiation dose, sex, and nodular sclerosis histology), the risk of hypothyroidism in white patients was 2.5 times (95% CI, 1.5 to 4.3 times) the risk in black patients (P < .001). CONCLUSION: White patients have a higher risk of hypothyroidism after neck irradiation for pediatric Hodgkin's lymphoma than black patients.

Adolescent↗

A simplified flow cytometric assay identifies children with acute lymphoblastic leukemia who have a superior clinical outcome.

Bone marrow normal lymphoid progenitors (CD19+, CD10+, and/or CD34+) are exquisitely sensitive to corticosteroids and other antileukemic drugs. We hypothesized that, in patients with B-lineage acute lymphoblastic leukemia (ALL), cells with this phenotype detected early in treatment should be leukemic rather than normal. We therefore developed a simple and inexpensive flow cytometric assay for such cells and prospectively applied it to bone marrow samples collected on day 19 from 380 children with B-lineage ALL. In 211 patients (55.5%), these cells represented 0.01% or more of the mononuclear cells; results correlated remarkably well with those of more complex flow cytometric and molecular minimal residual disease (MRD) evaluations. Among 84 uniformly treated children, the 10-year incidence of relapse or remission failure was 28.8% +/- 7.1% (SE) for the 42 patients with 0.01% or more leukemic cells on day 19 detected by the simplified assay versus 4.8% +/- 3.3% for the 42 patients with lower levels (P = .003). These assay results were the strongest predictor of outcome, even after adjustment for competing clinicobiologic variables. Thus, this new assay would enable most treatment centers to identify a high proportion of children with ALL who have an excellent early treatment response and a high likelihood of cure.

Antigens, CD19↗

Smaller white-matter volumes are associated with larger deficits in attention and learning among long-term survivors of acute lymphoblastic leukemia.

BACKGROUND: The primary objective of this study was to test the hypothesis that survivors of childhood acute lymphoblastic leukemia (ALL) have deficits in neurocognitive performance, and smaller white-matter volumes are associated with these deficits. METHODS: The patients studied included 112 ALL survivors (84 patients who had received chemotherapy only, 28 patients who had received chemotherapy and irradiation; 63 males, 49 females; mean age +/- standard deviation, 4.1 yrs +/- 2.6 yrs at diagnosis; mean +/- standard deviation yrs since diagnosis, 6.0 +/- 3.5 yrs), and 33 healthy siblings who participated as a control group. Neurocognitive tests of attention, intelligence, and academic achievement were performed; and magnetic resonance images were obtained and subsequently were segmented to yield tissue volume measurements. Comparisons of neurocognitive measures and tissue volumes between groups were performed, and the correlations between volumes and neurocognitive performance measures were assessed. RESULTS: Most performance measures demonstrated statistically significant differences from the normative test scores, but only attention measures exceeded 1.0 standard deviation from normal. Patients who had received chemotherapy alone had significantly larger volumes of white matter than patients who had received treatment that also included cranial irradiation, but their volumes remained significantly smaller than the volumes in the control group. Smaller white-matter volumes were associated significantly with larger deficits in attention, intelligence, and academic achievement. CONCLUSIONS: Survivors of childhood ALL had significant deficits in attention and smaller white-matter volumes that were associated directly with impaired neurocognitive performance. Cranial irradiation exacerbated these deficits.

Adolescent↗