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Archives of Endocrinology and Metabolism May 2024Hemangioblastomas associated with von Hippel-Lindau (VHL) disease are frequently multiple and recur during prolonged follow-up. Currently, no systemic treatment is... (Review)
Review
Expression of somatostatin receptors in hemangioblastomas associated with von Hippel-Lindau disease as a novel diagnostic, therapeutic, and follow-up opportunity: A case report and literature review.
Hemangioblastomas associated with von Hippel-Lindau (VHL) disease are frequently multiple and recur during prolonged follow-up. Currently, no systemic treatment is available for these tumors. Recent studies have shown the expression of somatostatin receptors in these types of hemangioblastomas. Notably, increased somatostatin receptor expression in a tumor, as determined by peptide-receptor radionuclide imaging, is a predictive factor of response to treatment with somatostatin analogs and peptide-receptor radionuclide therapy. The aim of this study was to describe the case of a patient with increased expression of somatostatin receptors in a suprasellar hemangioblastoma associated with VHL disease and conduct a literature review on somatostatin receptor expression in patients with VHL-associated hemangioblastomas. We describe herein the case of a 51-year-old man with VHL disease who had a suprasellar hemangioblastoma detected on magnetic resonance imaging. Peptide-receptor radionuclide imaging using gallium-68-DOTATOC (Ga-DOTATOC) identified increased expression of somatostatin receptors in the suprasellar hemangioblastoma, along with multiple pancreatic neuroendocrine tumors and bilateral pheochromocytomas. The patient was treated for 1 year with lanreotide, a somatostatin analog. A repeat Ga-DOTATOC 1 year after starting lanreotide revealed decreased radiotracer uptake by the hemangioblastoma, consistent with a metabolic response. The presence of somatostatin receptors in hemangioblastomas associated with VHL disease is a novel finding. The decreased expression of these receptors after treatment with a somatostatin analog, as described in the present case, positions the somatostatin receptor as a new target for novel diagnostic, therapeutic, and follow-up opportunities in patients with VHL disease.
Topics: Humans; Hemangioblastoma; von Hippel-Lindau Disease; Receptors, Somatostatin; Male; Middle Aged; Octreotide; Cerebellar Neoplasms; Follow-Up Studies; Magnetic Resonance Imaging; Radiopharmaceuticals
PubMed: 38788146
DOI: 10.20945/2359-4292-2023-0181 -
Liver Transplantation : Official... May 2024Hepatorenal syndrome-acute kidney injury (HRS-AKI) is associated with significant morbidity and mortality. While liver transplantation is the definitive treatment,...
BACKGROUND AND AIMS
Hepatorenal syndrome-acute kidney injury (HRS-AKI) is associated with significant morbidity and mortality. While liver transplantation is the definitive treatment, continuous terlipressin infusion for HRS-AKI may provide benefit and as such was assessed in a population comprised of liver transplant (LT) candidates.
METHODS AND RESULTS
Fifty hospitalized, LT-eligible patients with HRS-AKI received a single bolus followed by continuous terlipressin infusion. ACLF grade 3, serum creatinine (SCr)>5.0 mg/dL, or MELD≥35 were exclusions. Fifty hospitalized patients who received midodrine and octreotide (M&O) or norepinephrine (NorEpi) for HRS-AKI served as a historical comparator cohort. Complete response (CR) was defined as ≥30% decrease in SCr with EOT SCr≤1.5, partial response (PR) as ≥30% decrease in SCr with EOT SCr>1.5, and non-response (NR) as <30% decrease in SCr. CR rate was significantly higher in the terlipressin cohort compared to the historical cohort (64% vs. 16%, p<0.001). Survival, while numerically higher in those who received terlipressin, was statistically similar (D30: 94% vs. 82%, p=0.12; D90: 78% vs. 68%, p=0.37). Renal replacement therapy (RRT) was more common among terlipressin NR than CR and PR (70% vs. 3% vs. 13%, p<0.001). EOT MELD and SCr were significantly lower within terlipressin cohort (MELD: 19 vs. 25, SCr: 1.4 vs. 2.1 mg/dL, p<0.001). Sixteen of 40 terlipressin-treated patients received LT-alone (terlipressin CR in 10/16). One patient on terlipressin had hypoxic respiratory failure that responded to diuretics; one possibly had drug-related rash.
CONCLUSIONS
With continuous terlipressin infusion, a CR rate of 64% was observed with a favorable safety profile. Terlipressin use was associated with lower EOT MELD and SCr than the historical M&O/NorEpi cohort; LT-alone was accomplished in a high proportion of complete terlipressin responders.
PubMed: 38771635
DOI: 10.1097/LVT.0000000000000399 -
Clinical Nuclear Medicine Jul 2024This single-center retrospective study explores the safety and efficacy of 177 Lu-DOTATATE in children and young adult population with metastatic/inoperable...
PURPOSE
This single-center retrospective study explores the safety and efficacy of 177 Lu-DOTATATE in children and young adult population with metastatic/inoperable neuroendocrine tumors (NETs).
PATIENTS AND METHODS
This study is a retrospective analysis of all children and young adult patients (≤29 years) with advanced inoperable/metastatic epithelial or nonepithelial NETs who were administered a median of 4 cycles of 177 Lu-DOTATATE therapy and low-dose oral capecitabine as a radiosensitizer every 8-12 weeks, except 2 patients who received CAPTEM chemotherapy. The radiological response was assessed using RECIST 1.1 on interim and end-of-treatment 68 Ga-DOTANOC PET/CT. The primary endpoint was objective response rate, whereas disease control rate, toxicity profile, progression-free survival, and overall survival were secondary endpoints.
RESULTS
Nineteen biopsy-proven NET patients (median age, 22 ± 10 years) with 8 of them adolescents (10-18 years) and the remaining young adults (19-29 years) were included. Fourteen patients had gastroenteropancreatic neuroendocrine tumor (pancreas being most common primary site), whereas the rest had non-gastroenteropancreatic neuroendocrine tumor. A total of 65 cycles of 177 Lu-DOTATATE (range, 1-6 cycles) were administered with a median cumulative activity of 600 mCi (range, 100-1000 mCi). The objective response rate and disease control rate were 41% and 94%, respectively. Grade 1 and 2 adverse events were observed in 14 (74%) and 5 (26%) of 19 patients, respectively. In a total of 8 events (42%), 4 events each of disease progression and death occurred during a median follow-up of 80.1 months with an estimated 5-year progression-free survival and overall survival of 54% (95% confidence interval, 30-78) and 63% (95% confidence interval, 39-87), respectively.
CONCLUSIONS
177 Lu-DOTATATE appears safe and effective in children and young adults with metastatic/inoperable NETs. Large prospective trials are required to validate these results.
Topics: Humans; Organometallic Compounds; Adolescent; Male; Adult; Female; Young Adult; Child; Neuroendocrine Tumors; Retrospective Studies; Octreotide; Treatment Outcome; Safety
PubMed: 38769655
DOI: 10.1097/RLU.0000000000005233 -
Leukemia & Lymphoma May 2024
PubMed: 38767292
DOI: 10.1080/10428194.2024.2352085 -
Case Reports in Oncology 2024Lung neuroendocrine tumors (NETs) are a rare type of pulmonary tumor and represent approximately 2% of all lung cancers. The prevalence of lung NETs is increasing, which...
INTRODUCTION
Lung neuroendocrine tumors (NETs) are a rare type of pulmonary tumor and represent approximately 2% of all lung cancers. The prevalence of lung NETs is increasing, which may be due to improved diagnostic techniques for asymptomatic tumors. Diffuse idiopathic pulmonary neuroendocrine cell hyperplasia (DIPNECH) is a rare and underdiagnosed disease that falls under the spectrum of NETs.
CASE PRESENTATION
We presented a case of a 59-year-old male who presented with severe coughing spells, flushing, and diarrhea. His computed tomography scan showed innumerable pulmonary nodules and irregular nodular opacities throughout the lungs. He underwent a left upper lobe wedge resection and was eventually diagnosed with neuroendocrine tumorlets via immunohistochemical stains. He was started on a trial of octreotide and reported significant improvement in symptoms after 1 month.
CONCLUSION
DIPNECH is a rare preinvasive lesion characterized by the abnormal proliferation of pulmonary neuroendocrine cells. Patients with DIPNECH can present initially with respiratory symptoms, while other cases are discovered incidentally during the workup of different conditions. Definitive diagnosis of DIPNECH requires histopathological examination of lung tissue. There is limited evidence on DIPNECH management, and an individualized approach is currently advised.
PubMed: 38751830
DOI: 10.1159/000538796 -
PloS One 2024Peptide receptor radionucleotide therapy (PRRT) with 177Lu-dotatate is widely used for the treatment of patients with neuroendocrine tumors (NETs). We analyzed data from...
Peptide receptor radionucleotide therapy (PRRT) with 177Lu-dotatate is widely used for the treatment of patients with neuroendocrine tumors (NETs). We analyzed data from 104 patients with NETs treated with 177Lu -dotatate at a US academic center between December 2017 and October 2020 to better understand patterns of long-term efficacy, safety, and toxicity in the real-world setting. 177Lu-dotatate (200 mCi) was administered every eight weeks for four doses. The most common sites of primary disease were small intestine NETs (n = 49, 47%), pancreatic NETs (n = 32, 31%), and lung NETs (n = 7, 7%). Twenty-seven percent had Ki-67 <3%, 49% had Ki-67 between 3-20%, and 13.5% had Ki-67 >20%. The cohort had been pretreated with a median of two prior lines of treatment. Forty percent had received prior liver-directed treatment. Seventy-four percent of patients completed all four doses of treatment. The objective response rate was 18%. The median time-to-treatment failure/death was significantly longer for small-bowel NETs when compared to pancreatic NETs (37.3 months vs. 13.2 months, p = 0.001). In a multivariate model, Ki-67, primary site, and liver tumor burden ≥50% were found to independently predict time-to-treatment failure/death. Around 40% of patients experienced adverse events of ≥grade 3 severity. Treatment-related adverse events leading to discontinuation of therapy happened in 10% of patients. Preexisting mesenteric/peritoneal disease was present in 33 patients; seven of these patients developed bowel-related toxicities including two grade 5 events. We also report two cases of delayed-onset minimal change nephrotic syndrome, which occurred 14 and 27 months after the last dose of PRRT. Lastly, we describe six patients who developed rapid tumor progression in the liver leading to terminal liver failure within 7.3 months from the start of PRRT, and identify potential risk factors associated with this occurrence, which will need further study.
Topics: Humans; Neuroendocrine Tumors; Male; Female; Middle Aged; Aged; Octreotide; Receptors, Peptide; Adult; Treatment Outcome; Organometallic Compounds; Aged, 80 and over; Radiopharmaceuticals; Pancreatic Neoplasms; Retrospective Studies
PubMed: 38748739
DOI: 10.1371/journal.pone.0298824 -
Clinical Nuclear Medicine Jul 2024This case report explores the use of 177 Lu-DOTATATE in a hemodialysis patient. For the first time, this study assesses the average dose received by the bone marrow, the...
This case report explores the use of 177 Lu-DOTATATE in a hemodialysis patient. For the first time, this study assesses the average dose received by the bone marrow, the primary organ at risk, using an original double estimation method through independently acquired imaging and biological samples counting data. Despite elevated doses, the absorbed doses to the bone marrow (0.662-0.740 Gy) were within safe limits. Radiation protection measurements for staff were also compliant. This work supports that effective early dialysis and systematic personalized dosimetry are crucial for hemodialysis patients undergoing 177 Lu-PRRT due to their variability (residual excretion, treatment history, etc).
Topics: Humans; Renal Dialysis; Organometallic Compounds; Octreotide; Kidney Failure, Chronic; Radiometry; Male; Middle Aged; Radiotherapy Dosage; Female
PubMed: 38739496
DOI: 10.1097/RLU.0000000000005262 -
Clinical Nuclear Medicine Jul 2024In adults, 68 Ga-FAP inhibitor ( 68 Ga-FAPI) PET/CT outperforms 68 Ga-radiolabeled somatostatin analog peptides ( 68 Ga PET/CT) and 18 F-FDG PET/CT in detecting thyroid...
In adults, 68 Ga-FAP inhibitor ( 68 Ga-FAPI) PET/CT outperforms 68 Ga-radiolabeled somatostatin analog peptides ( 68 Ga PET/CT) and 18 F-FDG PET/CT in detecting thyroid lesions. This is the case of a 13-year-old boy newly diagnosed with medullary thyroid cancer with high calcitonin level. 68 Ga PET/CT revealed the presence of only a primary thyroid lesion. Proven to be superior in detecting metastasis, 68 Ga-FAPI PET/CT was performed. The results came out negative for primary and potential metastatic lesions. This case sheds shed light on false-negatives reported in 68 Ga-FAPI PET/CT scans in pediatric patients, emphasizing the need for alternate radiotracers when a negative study is met.
Topics: Humans; Male; Adolescent; Thyroid Neoplasms; Positron Emission Tomography Computed Tomography; Fluorodeoxyglucose F18; Carcinoma, Neuroendocrine; Organometallic Compounds; Octreotide; False Negative Reactions
PubMed: 38717245
DOI: 10.1097/RLU.0000000000005259 -
Cureus Apr 2024Beckwith-Wiedemann syndrome (BWS) is a rare genomic imprinting disorder that affects multiple systems. Major features can manifest as large birth weight, anterior...
Beckwith-Wiedemann syndrome (BWS) is a rare genomic imprinting disorder that affects multiple systems. Major features can manifest as large birth weight, anterior abdominal wall defects, macroglossia, hyperinsulinism, organomegaly hemihypertrophy, and renal abnormalities. Characteristic facies manifested as midface hypoplasia, infraorbital creases, facial nevus simplex, and anterior linear ear lobe creases/posterior helical ear pits, with a predisposition to tumor development. This case report describes a Saudi infant born at 38+5 weeks gestation via elective cesarean section to a 33-year-old G3P2+0 mother, with a family history of type 1 diabetes and Down syndrome. Prenatal ultrasound revealed an anterior abdominal wall defect. Postnatally, the infant exhibited macrosomia, macroglossia, and omphalocele. Genetic testing confirmed paternal disomy of the imprinted region in 11p15.5. The infant underwent successful omphalocele repair but experienced respiratory distress, and seizures on the third day of life. Intubation, ventilation, and antiepileptic treatment were initiated. Subsequent investigations revealed right upper lobe collapse, neonatal seizures on electroencephalogram (EEG), and thin corpus callosum on magnetic resonance imaging (MRI). Feeding difficulties led to elective partial glossectomy at two months of age. During her hospital stay two days post surgery, the infant developed persistent hypoglycemia requiring high glucose infusion rates. Extensive endocrine evaluation revealed high insulin and cortisol levels. Subcutaneous octreotide was administered with minimal response. After 15 days of careful glucose tapering, the infant's blood glucose stabilized, reaching feeding targets. The patient was discharged with follow-up appointments. This comprehensive case highlights the complexity of managing severe relapsing hypoglycemia in an infant with BWS.
PubMed: 38707113
DOI: 10.7759/cureus.57588 -
Trials May 2024
Correction: Methodology of the SORENTO clinical trial: a prospective, randomised, active-controlled phase 3 trial assessing the efficacy and safety of high exposure octreotide subcutaneous depot (CAM2029) in patients with GEP-NET.
PubMed: 38698434
DOI: 10.1186/s13063-024-08146-1