Pediatric Blood & Cancer最新文献

Background: Many preventive modalities have been advocated to reduce the incidence and severity of chemotherapy-induced Oral Mucositis (OM). Photobiomodulation, also known as Low-Level laser. Therapy, has shown promising results. This clinical trial was performed to evaluate the effectiveness of Photobiomodulation (PBM) for the prevention of chemotherapy-induced oral mucositis (OM) in children with Acute Myeloid Leukemia (AML).

Patients and methods: A double-blinded randomized clinical trial was performed, including forty-two children diagnosed with Acute Myeloid Leukemia (AML) admitted to receiving chemotherapy (CT) at Children's Cancer Hospital 57357, Cairo, Egypt. Patients were randomly assigned to either the control or test group. Both groups followed the standard preventive protocol for oral mucositis. In the control group I sham therapy, while in the Laser group I had Photobiomodulation sessions for five consecutive days once a day performed by Diode laser (Sirrolaser Blue ™, USA) 660nm, 15watt, 10 s. Laser sessions began on day 1 of chemotherapy and were repeated on days 2,3, 4, and 5. The response to both groups was evaluated according to the development of oral mucositis; graded using the classification criteria of the World Health Organization (WHO) and the National Cancer Institute scale (NCI - Common Terminology Criteria for Adverse Events, version 4.0). Incidence of oral mucositis was evaluated from baseline to 5, 12, 19, and 30 days after treatment.

Results: A clinical significant difference between the laser and control group was observed. The laser group showed a higher prevalence of no oral mucositis with decreased incidence of OM, while on day 12, the control group showed a higher prevalence of grades I and II (P-value <0.001, effect size = 0.796). However, the laser group showed higher prevalence of mucosa free of OM; on day 19, the control group showed higher rates of grades II and III, (P-value <0.001, effect size = 0.670). while on day 30 there was higher rates of grades I and II (P-value <0.001, effect size = 0.576) with higher rates of healthy oral mucosa in the laser group.

Conclusion: Photobiomodulation effectively reduces the incidence and severity of chemotherapy-induced oral mucositis in pediatric patients diagnosed with AML.

Background: ACKR1/DARC-associated neutropenia (ADAN), resulting from homozygosity for a single nucleotide polymorphism (SNP) in the ACKR1/DARC gene (rs2814778), is a common cause of benign neutropenia that primarily affects individuals of African and Jewish Yemenite descent. We aimed to characterize ADAN in pediatric patients in Israel, given its ethnically diverse population.

Procedure: We assessed children with isolated neutropenia treated during 2018-2023 at one pediatric center, for the ACKR1/DARC polymorphism, using Sanger sequencing or targeted next-generation sequencing.

Results: Of 115 patients evaluated, 49 (42.6%) were diagnosed with ADAN; of these, 29 (59%) had absolute neutrophil counts in the severe range (0-0.5 × 10⁹/L) at diagnosis. The allele distribution revealed 37% of Muslim Arab and 61% of Jewish origin. Yemenite, Ethiopian, Mediterranean, Asian, and European ancestry were included; 59% had a family history of neutropenia. The median age at the first neutropenia detection was 1.2 years; 91.8% were identified during routine blood counts. The median absolute neutrophil count at diagnosis was 0.5 × 10⁹/L (interquartile range: 0.3). An increased susceptibility to infections was not found either before or during the median follow-up period of 2.5 years (interquartile range: 1.54) after the diagnosis of ADAN. In 34 patients (72.3%), neutrophil counts were in the normal range during febrile illnesses.

Conclusions: We identified ADAN in individuals of variable ethnicities, almost half with severe neutropenia. We recommend testing for ADAN in all children with isolated neutropenia without severe infections. Homozygosity for the ACKR1/DARC rs2814778 SNP may obviate the need for further investigation, follow-up, or treatment in specific clinical scenarios.

Purpose: Platinum-based chemotherapy is a mainstay of treatment for many childhood cancers but is associated with acute nephrotoxicity and long-term ototoxicity. There is emerging evidence of long-term renal complications. This study aimed to assess the prevalence of chronic kidney disease (CKD) in children treated with platinum chemotherapy (cisplatin and carboplatin) and identify potential risk factors for the development of CKD.

Methods: We conducted a retrospective review of children diagnosed with hepatoblastoma, osteosarcoma, neuroblastoma, or medulloblastoma who received platinum chemotherapy over a 16 year timeframe. Patients were excluded if they did not have at least 3 years follow up data, died within 3 years of platinum chemotherapy, or if they relapsed. Clinical data were collected at baseline (first dose), 1 year, and at most recent follow up.

Results: Of 328 treated patients, 147 met the inclusion criteria and were followed for a mean of 8.1 years (range 3-15.7 years). The median age at first dose was 3.7 years (IQR 1.7-9.6 years). CKD ≥grade 2 was present in 53(36%) at last follow up and 15(10%) had tubular dysfunction. A history of acute kidney injury at any time during treatment was associated with CKD (OR 3.12 CI 1.07-9.12, p =0.04). On multivariable analysis older age at platinum therapy (OR 1.2, CI 1.1-1.4, p = 0.004) and a high aminoglycoside or vancomycin trough level (OR 4.3, CI 1.9-9.7, p < 0.001) were risk factors for CKD.

Conclusion: The high rate of CKD in children treated with platinum chemotherapy warrants long-term follow-up and screening for progressive disease.

Background: Veno-occlusive disease (VOD) is a life-threatening endotheliopathy that can occur after stem cell transplant (SCT). Numerous risk factors contribute to the development of VOD during SCT, and the role of prophylactic defibrotide (DF) in mitigating these risks remains unclear.

Objective: We compare not only the incidence of VOD development, but also the severity of VOD and survival outcomes between patients who did and did not develop VOD and did or did not receive prophylactic DF.

Study design: In this single-center retrospective study of 58 pediatric SCT patients from 2008 to 2022, we compare the demographics, risk profiles, and outcomes within three cohorts: Group 1: prophylactic DF and no VOD (n = 5), Group 2: prophylactic DF and development of VOD (n = 6), and Group 3: treatment DF for patients who developed VOD (n = 47).

Results: Patients with VOD who did not receive prophylactic DF had higher severity classification of disease at onset (very severe 80.9% vs. 66.7%, p = .592) and at maximum severity (very severe 89.4% vs. 83.3%, p = .532), as opposed to mild, moderate, or severe categorization compared to those who did not receive prophylactic DF. Patients who developed VOD and did not receive prophylactic DF had a lower 1-year survival probability compared to those who received prophylactic DF and still developed VOD (51.1% vs. 75% alive at 1 year, excluding the two subjects without adequate follow-up time, p = .266).

Conclusion: Although, not statistically significant in our small retrospective study, there is potential overall survival and decreased VOD severity benefits of prophylactic DF.