“…Treatment was discontinued due to patient request ( n = 10), disease progression ( n = 2), and adverse events ( n = 0). One of the enrolled patients was previously reported in a BJOG case report [ 16 ], but in this study, the patient’s medication and follow-up time were extended to 54 months. Fig.…”
Background
Intravenous leiomyomatosis (IVL), pulmonary benign metastatic leiomyomatosis (PBML), and leiomyomatosis peritonealis disseminata (LPD) are leiomyomas with special growth patterns and high postoperative recurrence rates. We report the safety and efficacy of a pilot study of sirolimus in the treatment of recurrent IVL, PBML, and recurrent LPD.
Methods
This was a pilot study to evaluate the safety and efficacy of sirolimus in the treatment of leiomyomatosis (ClinicalTrials.gov identifier NCT03500367) conducted in China. Patients received oral sirolimus 2 mg once a day for a maximum of 60 months or until disease progression, intolerable toxicity, withdrawal of consent, or investigator decision to stop. The primary end point of this study was the objective response rate. Secondary end points included safety and tolerability, disease control rate, and progression-free survival.
Results
A total of 15 patients with leiomyomatosis were included in the study, including five with recurrent IVL, eight with PBML and two with recurrent LPD. The median follow-up time was 15 months (range 6–54 months), nine patients (60%) had treatment-related adverse events (including all levels), and two patients had treatment-related grade 3 or 4 adverse events. The objective response rate was 20.0% (95% CI, 7.1–45.2%), and the disease control rate was 86.7% (95% CI, 62.1–96.3%). Partial response was achieved in three patients. The median response time in the three partial response patients was 33 months (range 29–36 months), and the sustained remission time of these three patients reached 0, 18, and 25 months, respectively.
Conclusions
Sirolimus was safe and effective in the treatment of recurrent IVL, PBML, and recurrent LPD.
Trial registration
ClinicalTrials.gov identifier NCT03500367. Registered on 18 April 2018.
“…Treatment was discontinued due to patient request ( n = 10), disease progression ( n = 2), and adverse events ( n = 0). One of the enrolled patients was previously reported in a BJOG case report [ 16 ], but in this study, the patient’s medication and follow-up time were extended to 54 months. Fig.…”
Background
Intravenous leiomyomatosis (IVL), pulmonary benign metastatic leiomyomatosis (PBML), and leiomyomatosis peritonealis disseminata (LPD) are leiomyomas with special growth patterns and high postoperative recurrence rates. We report the safety and efficacy of a pilot study of sirolimus in the treatment of recurrent IVL, PBML, and recurrent LPD.
Methods
This was a pilot study to evaluate the safety and efficacy of sirolimus in the treatment of leiomyomatosis (ClinicalTrials.gov identifier NCT03500367) conducted in China. Patients received oral sirolimus 2 mg once a day for a maximum of 60 months or until disease progression, intolerable toxicity, withdrawal of consent, or investigator decision to stop. The primary end point of this study was the objective response rate. Secondary end points included safety and tolerability, disease control rate, and progression-free survival.
Results
A total of 15 patients with leiomyomatosis were included in the study, including five with recurrent IVL, eight with PBML and two with recurrent LPD. The median follow-up time was 15 months (range 6–54 months), nine patients (60%) had treatment-related adverse events (including all levels), and two patients had treatment-related grade 3 or 4 adverse events. The objective response rate was 20.0% (95% CI, 7.1–45.2%), and the disease control rate was 86.7% (95% CI, 62.1–96.3%). Partial response was achieved in three patients. The median response time in the three partial response patients was 33 months (range 29–36 months), and the sustained remission time of these three patients reached 0, 18, and 25 months, respectively.
Conclusions
Sirolimus was safe and effective in the treatment of recurrent IVL, PBML, and recurrent LPD.
Trial registration
ClinicalTrials.gov identifier NCT03500367. Registered on 18 April 2018.
“…The obstetrics and gynecology team at Beijing Union Medical College Hospital endeavored to employ rapamycin (sirolimus) as a treatment modality for a recurrent patient who was deemed unsuitable for additional surgical intervention. Encouragingly, subsequent monitoring demonstrated a noteworthy decrease in the lesion ( 45 ).…”
ObjectiveIntravenous leiomyomatosis (IVL) is a rare and aggressive tumor type that has the potential to extend into the inferior vena cava (IVC) and is susceptible to be misdiagnosed and neglected. Despite its clinical significance, there is a paucity of research that has focused on the specific manifestations of IVL on ultrasonography. Therefore, this study aims to systematically analyze the specific ultrasound features of IVL and augment its diagnostic accuracy.Materials and methodProspective inclusion was granted to patients admitted to our hospital between December 2016 and March 2021 for an IVC-occupying lesion. Multi-modal ultrasonography, encompassing gray-scale and color Doppler, was conducted. Lesions were categorized as IVL or non-IVL based on pathological or follow-up data. Two ultrasound sonographers with over 5 years of experience read and recorded ultrasound data for all lesions, which were subsequently comparatively analyzed to identify specific signs of IVL.ResultsA total of 284 patients diagnosed with IVC-occupying lesions were included in the study. The lesion types comprised of IVL (n=67, 23.6%), IVC thrombus (n=135, 47.5%), tumor thrombus of renal carcinoma involving the IVC (n=35, 12.4%), tumor thrombus of liver carcinoma involving the IVC (n=24, 8.5%), leiomyosarcoma of the IVC (n=14, 4.9%), and tumor thrombus of adrenocortical adenocarcinoma (n=9, 4.1%). The presence of “sieve hole” and “multi-track” signs was observed in 20 IVL lesions under the grey-scale modality, while both signs were absent in the non-IVL group (P<0.01). The study found no statistically significant differences in the presentation of “sieve hole” and “multi-track” signs under the grey-scale and color Doppler modalities in cases of intravascular lithotripsy (IVL) (P>0.05). Using these two signs as diagnostic criteria for IVL, the sensitivity, specificity, positive predictive value (PPV), negative predictive value (NPV), miss rate, misdiagnosis rate, and accuracy were determined to be 29.9%, 100%, 100%, 82.2%, 70.1%, 0, and 83.5%, respectively (AUC ROC=0.649; 95%CI: 0.537-0.761).ConclusionIVL exhibits distinct ultrasound presentations, including “sieve hole” and “multi-track” signs, which demonstrate high specificity and accuracy as diagnostic indicators. Furthermore, these signs are corroborated by pathological evidence and effectively distinguish IVL from other lesions occupying the IVC.
“…In another disease also caused by activation of mTOR signaling pathway and characterized by smooth muscle cell in ltration, lymphangioleiomyomatosis, rapamycin has showed persistent e cacy and long-term safety [14]. Furthermore, Zhang et al recently reported the rst successful control of disease with rapamycin in treating intravenous leiomyomatosis [15]. In Case 1, we also obtained WES data from PBML and UL samples.…”
Background: Pulmonary benign metastasizing leiomyoma (PBML) is the most common extrauterine spread of uterine leiomyoma and its biological behavior is traditionally thought to be hormone-dependent. Studies about the older PBML patients has been previously reported, but limited literature has been regarding to the clinical features and treatment of PBML in young women.
Methods: A total of 65 cases of PBML in women aged 45 years and younger were reviewed, including 56 cases selected from PubMed and 9 cases from our hospital. The clinical characteristics and management of these patients were analyzed.
Results: The median age of all the patients at diagnosis was 39.0 years. PBML most commonly presented as bilateral solid lesions (60.9%), with other rare imaging manifestations. Median interval time from a pertinent gynecologic procedure to diagnosis was 6.0 years. 16.7% of patients received careful observation and all achieved stable status in a median follow-up time of 18.0 months. 71.4% of patients were administrated anti-estrogen therapies, including surgical castration (33.3%), gonadotropin-releasing hormone analogue (23.8%) and anti-estrogen drugs (14.3%). Eight of 42 patients underwent surgical resection of metastatic lesions. Patients receiving curative surgery for the removal of pulmonary lesions, combined with adjuvant anti-estrogen therapies, had favorable outcomes compared with the ones who only received surgical resection. The disease control rates of surgical castration, gonadotropin-releasing hormone analogue, anti-estrogen drugs were 85.7%, 90.0%, 50.0%, respectively. Rapamycin for two patients achieved successful relief of symptoms and control of pulmonary lesions without lowering hormone levels and causing estrogen deficiency symptoms.
Conclusions: In the absence of standard treatment guidelines for PBML, maintaining a low-estrogen environment by different kinds of anti-estrogen therapies has been the mainstream strategy currently and has satisfying curative effects. A wait-and-see strategy might be an option, but therapeutic approaches must be contemplated when complications or symptoms progressed. For PBML in young women, the negative effect on ovarian function of anti-estrogen treatment especially surgical castration should be considered. Rapamycin might be a new treatment option for young PBML patients, especially for those ones who want to preserve ovarian function.
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