Pediatric Hematology and Oncology最新文献

Transient abnormal myelopoiesis (TAM) is a unique disease occurring in Down syndrome (DS) infants from which most patients have spontaneous remission. This study aimed to evaluate the incidence and outcomes of TAM in a tertiary center in Thailand. We reviewed the records of 997 DS patients diagnosed between June 1993 and October 2019. From the 997 DS patients, 32 had been diagnosed with TAM. The incidence of TAM was 3.2% and an overall survival rate of 87.5%. A total of 2/28 who survived (7.1%) subsequently developed AML-DS at the ages of 2.1 and 4.5 years, respectively. The risk factors related with death included maternal multiparity, sepsis, skin bleeding, subcutaneous nodules, high WBC count, low hemoglobin, and elevated AST level.Abbreviations.

Inborn errors of immunity (IEI) are a group of disorders caused by genetically determined defects in the immune system, leading to infections, autoimmunity, autoinflammation and an increased risk of malignancy. In some cases, a malignancy might be the first sign of an underlying IEI. As therapeutic strategies might be different in these patients, recognition of the underlying IEI by the pediatric hemato-oncologist is important. This article, written by a group of experts in pediatric immunology, hemato-oncology, pathology and genetics, aims to provide guidelines for pediatric hemato-oncologists on how to recognize a possible underlying IEI and what diagnostic tests can be performed, and gives some consideration to treatment possibilities.

Risk stratification of pediatric febrile neutropenia (FN) is an established concept, yet clinical risk tools misclassify nearly 5% of clinical standard-risk episodes with severe outcomes. The internal evaluation of a clinical risk tool before implementation has not been well-described. In this noninterventional cohort study, we evaluated a study decision rules (SDR) tool; a clinical risk tool with serial procalcitonin. The study standard-risk (SSR) group met clinical standard-risk criteria with two serial procalcitonin <0.4 ng/mL. The study high-risk (SHR) group met clinical high-risk criteria or clinical standard-risk with a procalcitonin ≥0.4 ng/mL. Descriptive and bivariate statistics compared the groups and outcomes. Clinical criteria alone identified 39.1% (238/608) standard-risk episodes; 5.9% (14/238) had severe events. Prospectively using the SDR, the SHR group encompassed 76.6% (92/120) of episodes; severe events occurred in 20% (3/15) of standard-risk episodes included due to elevated procalcitonin ≥0.4 ng/mL. The SHR group had more blood stream infections [21.7% (20/92) vs. 0% (0/28); P = 0.007] and intensive care admissions [13% (12/92) vs. 3.6% (1/28); P = 0.158]. In conclusion, the SDR with serial procalcitonin aided in identifying severe events in clinical standard-risk episodes, but analysis was limited. Institutions may consider similar internal evaluation methodology before FN episode risk stratification.

The COVID-19 pandemic has had a huge effect all over the world and its impact has been even more profound in the world of Healthcare. In this brief report we'd like to report about our experience in pediatric newly diagnosed thrombocytopenia and how we have seen the landscape of this disease change in the last 2 years. In particular, we believe that the use of personal protective equipment and lockdown measures have reduced the incidence of viral triggers that are supposed to be responsible for the vast majority of ITP cases. Along with these data, we found some other significant differences in the period taken into account.

CHG-based hygiene methods are often a component of daily hygiene bundles to prevent central line-associated blood stream infections (CLABSIs) in pediatric hematology-oncology patients; however, adherence with 2% CHG wipes was inconsistent within our institution, risking infection for immunocompromised patients. A new 4% CHG foam method offers an alternative and is applied while bathing, as opposed to wipes used 1 h after bathing. An initial cohort of 24 high-risk oncology and bone marrow transplant (BMT) patients agreed to use 4% CHG foam in place of wipes, and then answered surveys to describe their experiences. Ninety-two percent preferred foam over wipes and were more likely to use the foam moving forward. CHG foam was then made available as an option to all patients in need of central line care upon admission to the hospital. Hygiene bundles in the electronic medical record were reviewed to measure baseline adherence rates. Random audits by nursing administration prospectively assessed CHG adherence. CLABSI data were collected prospectively with routine quality metric reports. Results were analyzed using run charts and u-charts, respectively. Hematology-Oncology unit adherence rates remained at a higher rate of adherence, and BMT unit adherence rates increased from an average of 55%-81.6% (p < 0.001). Primary CLABSIs remained rare events (average <1/1000 CVL days). On cost analysis, utilizing CHG foam results in an annual savings estimate of $40,000 for a 24-bed unit. In conclusion, 4% CHG foam provides a cost-effective and patient-preferred option for daily hygiene that maintains CLABSI preventative efforts.

Data on childhood acute promyelocytic leukemia (APL) from low-and middle-income countries is limited. Early mortality is a concern and often not highlighted in clinical trials. The retrospective study was conducted on patients (≤12 years) with APL from 2003 to 2021 at a single center in India. Patients were treated with all-trans-retinoic acid (ATRA) and chemotherapy. Induction and three courses of consolidation were followed by maintenance for 2 years. In 2015, the protocol was updated with following modifications: (a) obtaining diagnostic cerebrospinal fluid at end-of-induction rather than at diagnosis, (b) administering intrathecal cytarabine regardless of risk-category, (c) risk-stratified administration of chemotherapy, and (d) inclusion of ATRA in all the cycles of consolidation. Sixty-two patients were diagnosed over the 17 years. The median age was 8 years (range: 0.9-12). Half had high-risk disease. Differentiation syndrome was observed in 32%, none being fatal. Eighteen (29%) patients died due to hemorrhage (83%) or septicemia (17%). Thirteen (21%) had early mortality (≤15 days), all due to hemorrhage. A platelet count <20 × 10⁹/L predicted early mortality (odds ratio: 4.5; 95% CI: 0.9-22, p = 0.06). Treatment abandonment reduced from 23.5% during 2003-2015 to nil during 2015-2021 (p = 0.006). Three (8%) patients relapsed. The 4-year OS of all patients and the patients who survived >15 days was 70.1% and 89.6%, respectively. The 4-year EFS, including abandonment and early mortality, before and following updated protocol, was 61.4% and 65.5%, respectively (p = 0.77). Early mortality continues to be a barrier to an otherwise excellent survival in childhood APL. A significant reduction in treatment abandonment in recent years is gratifying.

Pediatric central nervous system tumor survivors (CNSTS) experience late effects that may affect their health-related quality of life (HRQOL). The study aims: i) compare HRQOL among Malaysian CNSTS with acute lymphoblastic leukemia survivors (ALLS) and healthy children, and ii) explore factors associated with low HRQOL. We performed a comparative cross-sectional HRQOL study of 46 CNSTS aged 5-18 years and 90 ALLS (age and gender-matched) who completed treatment for >1 year, and a published cohort of healthy children. Pediatric Quality of Life Inventory (PedsQL) was used for all groups and PedsQL Cancer Module for CNSTS and ALLS. Multiple regression analysis was used to determine factors associated with low HRQOL. Mean PedsQL total scale score, physical health score and psychosocial health score of CNSTS were 69.0 (SD 20.3), 68.7 (SD 27.9) and 69.2 (SD 19.2) respectively. These scores were significantly lower in all domains particularly in teenagers compared with healthy children and ALLS. The median PedsQL Cancer Module score of CNSTS was significantly lower than ALLS in total scale, cognitive problems and communication. Physical impairment was associated with lower PedsQL scores in all 3 domains; special education placement was associated with lower PedsQL total scale and physical health scores and clinically significant internalizing behavioral difficulties score was associated with lower PedsQL psychosocial health scores. CNSTS reported lower PedsQL scores in all domains than ALLS and healthy children. Clinicians need to be vigilant of HRQOL needs among CNSTS, especially those with risk factors of special education needs, physical impairment, and internalizing behavioral difficulties.

Children with sickle cell anemia (SCA) usually face psychological complications especially depression. Assessment of depression in resource-limited settings may help identify the extent to which the children with SCA in such settings may need its introduction as part of routine care. This study aimed to assess depression in children and adolescents with SCA in a low-resource setting. This cross-sectional observational study involved 84 children and adolescents with SCA aged 7-17 years who were selected using a systematic random sampling technique. Their controls were 84 age- and sex-matched individuals with AA hemoglobin genotype. A structured questionnaire was used to collect socio-demographic data while depression was assessed with the Children's Depression Inventory. The prevalence of depression was non-significantly higher in subjects compared to the controls (8.3% vs. 2.4%) (Fisher's χ² = 1.88, p = 0.171). Though not statistically significant, the subjects had 3.7 times higher odds of having depression compared to the controls (OR = 3.7; 95% CI 0.75-18.50; p = 0.107). Of the 5 depression subscales, the subjects had a significantly higher difference in the negative mood (p = 0.042). Despite the comparable prevalence of depression with their normal controls, children and adolescents with SCA had a higher negative mood and higher odds of having depression than normal individuals. Thus, there is a need for the introduction of depression assessment as a complement to routine care of these children with SCA in resource-poor settings.

The enzyme phosphoglycerate kinase 1 (PGK1) catalyzes the first ATP producing reaction in the glycolysis pathway. Certain mutations to the coding gene of PGK1 present clinically with varying manifestations including hemolytic anemia, central nervous system (CNS) dysfunction and myopathy. Various PGK1 mutations have been described in the literature at the clinical and molecular level. Herein, we describe a novel case PGK1 mutation (PGK1 Galveston) in a 4-year-old boy who presented with all three manifestations. We discuss the characteristic hematopathology findings from this patient as well as provide a comparison with previously described neuroimaging findings. The variable clinical presentation of this condition along with its inherent uniqueness provide a diagnostic challenge for physicians. This presentation will add to the current body of knowledge for this condition and help guide future investigation and management.

There is significant debate over whether phase 1 pediatric oncology trials are ethical and approvable. We thus surveyed IRB members to answer four questions. First, do IRB members think the potential medical benefits of average phase 1 pediatric oncology trials justify the risks? Second, do they think these trials are ethically appropriate? Third, do they think these trials are approvable? Fourth, how do the views of IRB members on the first two questions compare to the views of the US public? Of the 80 respondents who answered the test questions correctly, 18.8% stated that the potential medical benefits of average phase 1 pediatric oncology trials outweigh the risks, 32.5% stated that the potential medical benefits and risks are about equal, and 48.8% stated that the risks outweigh the potential medical benefits. Compared to the general public, IRB members were significantly more likely to think the risks outweigh the potential medical benefits (p = 0.01). Finally, 68.8% of IRB members indicated that average phase 1 pediatric oncology trials are approvable, and 56.3% indicated that these trials are appropriate in children. These findings suggest two-thirds of IRB members believe average phase 1 pediatric oncology trials are approvable. Yet, almost half regard the risks as outweighing the potential medical benefits and almost half think these trials are inappropriate. These findings raise important questions regarding why IRB members and the general public evaluate the same risk/benefit profile differently, and whether it is possible to reconcile the two perspectives.