Health-Related Quality of Life in Children With Kaposiform Hemangioendothelioma

et al. 2023

Intl Journal of Cancer

Self Cite

Treatment with sirolimus, an inhibitor of the mammalian target of rapamycin pathway, has improved the prognosis of patients with kaposiform hemangioendothelioma (KHE). However, the efficacy, durability and tolerability of long-term sirolimus treatment in patients with KHE have not been well elucidated. We performed efficacy and safety assessments based on more than 4.5 years of follow-up in patients receiving sirolimus therapy for KHE. One hundred sixtyseven patients were analyzed, including 102 (61.1%) patients with the Kasabach-Merritt phenomenon (KMP). Follow-up was conducted after a median of 56.0 months. A total of 154 (92.2%) patients had a durable response to sirolimus treatment. No difference in durable response was found between patients without KMP and patients with KMP (95.4% vs 90.2%; difference, 5.2%; 95% confidence interval [CI], À4.0% to 13.1%). Rebound growth occurred in 17.3% of patients upon sirolimus discontinuation. Early treatment discontinuation (odds ratio [OR]: 3.103; 95% CI: 1.529-6.299; P = .002) and mixed lesion type (OR: 2.271; 95% CI: 0.901-5.727; P = .047) were associated with tumor rebound growth. No KHE-related deaths occurred in this cohort. At the last follow-up, approximately 17.4% of patients had active disease and/or changes in body structures to a variable extent. Serious adverse events occurred most commonly during the first year of sirolimus therapy. Follow-up of almost 4.5 years demonstrated that the efficacy of sirolimus persisted over time and that long-term treatment Abbreviations: CI, confidence interval; KHE, kaposiform hemangioendothelioma; KMP, Kasabach-Merritt phenomenon; MRI, magnetic resonance imaging; mTOR, mammalian target of rapamycin; OR, odds ratio; ROM, range of motion.Jiangyuan Zhou, Yanan Li and Tong Qiu have contributed equally to this work.

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Long‐term outcomes of sirolimus treatment for kaposiform hemangioendothelioma: Continuing successes and ongoing challenges

Zhou

et al. 2023

Intl Journal of Cancer

Self Cite

“… 4 , 10 In addition, KMP can affect the health-related quality of life of patients of all ages. 11 Meanwhile, recent research has shown that KMP patients require a longer duration of sirolimus treatment and have a greater rebound rate and more disease sequelae than KHE patients. 12 Given the poor prognosis of the condition and sequelae, KHE patients with KMP tend to require more aggressive management in clinical practice than KHE patients without KMP.…”

Section: Introductionmentioning

confidence: 99%

Impact of age and tumor size on the development of the Kasabach–Merritt phenomenon in patients with kaposiform hemangioendothelioma: a retrospective cohort study

Zhou

Lan

Precision Clinical Medicine

et al. 2023

Self Cite

Introduction The Kasabach-Merritt phenomenon (KMP) is a severe complication of kaposiform hemangioendothelioma (KHE). The risk factors for KMP need further investigation. Methods The medical records of patients with KHE were reviewed. Univariate and multivariate logistic regression models were used for the risk factors for KMP, and the area under the receiver operator characteristic (ROC) curve was used to assess the predictive power of risk factors. Results Three hundred thirty-eight patients with KHE were enrolled. The incidence of KMP was 45.9%. Age of onset (P < 0.001, odds ratio [OR] 0.939; 95% confidence interval [CI] 0.914–0.966), lesion size (P < 0.001, OR 1.944; 95% CI 1.646–2.296), mixed type (P = 0.030, OR 2.428; 95% CI 1.092–5.397), deep type (P = 0.010, OR 4.006; 95% CI 1.389–11.556), and mediastinal or retroperitoneal lesion location (P = 0.019, OR 11.864; 95% CI 1.497–94.003) were correlated with KMP occurrence through multivariate logistic regression. ROC curve analysis revealed that the optimal cutoffs were 4.75 months for the age of onset (P < 0.001, OR 7.206, 95% CI 4.073–12.749) and a lesion diameter of 5.35 cm (P < 0.001, OR 11.817, 95% CI 7.084–19.714). Bounded by a lesion size of 5.35 cm, we found significant differences in tumor morphology, age of onset, treatments and hematological parameters. Using an onset age of 4.75 months as a cutoff, we found significant differences in tumor morphology, lesion size, hematological parameters and prognosis. Conclusion For KHE patients with an onset age < 4.75 months and/or lesion diameter > 5.35 cm, clinicians should be wary of the occurrence of KMP. Active management is recommended to improve the prognosis.

“…Growth or expansion of VAs can lead to clinical problems, such as disfigurement, bleeding, recurrent infection, chronic pain, thrombocytopenia, consumptive coagulopathy, organ dysfunction, and death [ 2 – 5 ]. Patients with complicated VAs may suffer from progressive clinical symptoms with decreased quality of life [ 6 ]. Because of the broad spectrum and insufficient treatment regimens, the management of complicated VAs is complex and usually needs a multidisciplinary team with a combination of surgical, interventional (e.g., embolization), and medical treatments [ 7 , 8 ].…”

Section: Introductionmentioning

confidence: 99%

Oral antibiotic prophylaxis for infection in patients with vascular anomalies receiving sirolimus treatment: a multicenter retrospective study

Gong

et al. 2023

Orphanet J Rare Dis

Self Cite

Objectives Patients with vascular anomalies (VAs) who receive oral sirolimus may be at high risk of infectious complications. Antibiotic prophylaxis with trimethoprim-sulfamethoxazole (TMP-SMZ) has been advocated. However, there have been few evidence-based analyses on this topic. This study assessed the effect of prophylactic TMP-SMZ on the incidence of infections in VA patients receiving sirolimus monotherapy. Methods A retrospective, multicenter chart review was performed on all VA patients receiving sirolimus treatment from August, 2013 to January, 2021. Results Before January 2017, 112 patients were treated with sirolimus without antibiotic prophylaxis. In the subsequent period, 195 patients were treated with TMP-SMZ for at least 12 months during sirolimus therapy. The percentage of patients with at least one serious infection during the initial 12 months of sirolimus treatment did not differ between the groups (difference, 1.1%; 95% CI − 7.0–8.0%). We observed no difference in the incidence of individual infection or total adverse events between the groups. The rate of sirolimus discontinuation due to adverse events did not differ significantly between groups. Conclusions We demonstrated that prophylactic TMP-SMZ does not decrease the incidence of infection or improve tolerance in VA patients receiving sirolimus monotherapy.