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Table 2 Characteristics of patients treated with sirolimus

From: The impact of sirolimus therapy on lesion size, clinical symptoms, and quality of life of patients with lymphatic anomalies

N Age at start/ sex Diagnosis Location of target lesions Complications Previous treatment Dosing period (months) Range (mean) of trough concentrations (ng/mL) Evaluation of radiological volumetric change (change rate, %) Adverse effects associated with sirolimus (CTCAE Grade)
1 1 year/M Cystic LM Neck and tongue Disturbance of swallowing, dysarthria, lymphorrhea, and airway obstruction Sclerotherapy, Chinese herb 18 (cessation for surgery) 6.0–11.8 (7.6) PR (−28.1) Upper respiratory infection (3)
2 2 weeks/F Cystic LM Right neck, axilla, trunk, and abdominal cavity Anemia, coagulation disorder, and bleeding Blood transfusion 14 (cessation) 3.5–11.1 (6.2) PR (−27.9) None
3 3 years/F Cystic LM Left orbit Ocular displacement Steroids and propranolol 6 5.4–11.0 (8.3) PR (−24.3) None
4 10 months/F Cystic LM Neck and mediastinal Airway obstruction and disturbance of swallowing Sclerotherapy, Chinese herb 6 3.5–11.5 (7.2) PR (−23.4) Cellulitis (3)
5 11 years/M Cystic LM Neck and mediastinal Airway obstruction and disturbance of swallowing Sclerotherapy, Chinese herb 6 1.8–5.9 (3.9) SD (−15.0) Stomatitis (1)
6 8 years/M KLA Bone, thoracic and mediastinal Chylothorax and coagulation disorder Interferon and propranolol 30 (cessation) 4.4–9.0 (7.5) PR (−55.4) Pneumonia (3)
7 8 years/M KLA Bone, thoracic and mediastinal Scoliosis, chylothorax and coagulation disorder Steroids and propranolol 24 8.1–12.4 (11.2) SD (−8.3) Stomatitis (1)
8 20 years/M KLA Bone, thoracic and right chest wall Gastrointestinal hemorrhage and coagulation disorder Steroids 12 4.7–6.0 (5.5) PR (−48.4) Stomatitis (1)
9 32 years/F GLA Abdominal cavity and skin Ascites, coagulation disorder, and lymphorrhea Chinese herb 6 3.1–9.8 (6.2) PR (−76.5) None
10 13 years/F GLA Left upper limb, spleen and skin Lymphorrhea, bleeding and cellulitis Surgery, Chinese herb 8 2.7–7.2 (5.0) SD (−0.1) Stomatitis (1)
11 35 years/F GLA Abdominal cavity and skin Lymphorrhea, pain and cellulitis Surgery, Chinese herb 7 (cessation) 8.3–13.8 (11.0) SD (−1.0) Stomatitis (1)
12 20 years/F GSD Right thigh bone Pathological fracture and pain Surgery 18 (cessation) 3.2–10.0 (5.8) PR (− 59.9) Stomatitis (1)
13 9 years/M GSD Skull base Hearing loss and spinal fluid leakage Bisphosphonate, interferon, and propranolol 18 (cessation) 3.6–6.2 (5.7) SD (−19.1) Feeling of fatigue (1)
14 20 years/M GSD Right lower limbs Lymphorrhea and spinal nerve palsy Bisphosphonate, interferon, epidural blood patch therapy 6 (cessation) 3.0–10.9 (6.9) PR (−20.6) Cellulitis (3)
15 39 years/M GSD Paranasal sinus Facial nerve palsy, pain, cerebral infarction, skull base osteomyelitis, and lateral medullary syndrome Bisphosphonate and radiotherapy 6 (death from progression of disease) 3.5–7.4 (6.7) SD (−7.1) Stomatitis (1)
16 22 years/M GSD Right mandible Spinal fluid leakage, pain, and mal interdigitation Surgery 7 3.5–11.6 (7.0) PR (−52.3) None
17 18 years/M GSD Right thigh, lower limbs Lymphorrhea and pain Surgery, Bisphosphonate, interferon, Chinese herb, and propranolol 14 3.2–5.1 (4.2) SD (−2.9) Stomatitis (1)
18 3 years/F CCLA Thoracic and mediastinal Appendicular lymphedema, respiratory disorder, dyspnea, and wheeze Steroids and lymphatic venous anastomosis 24 3.1–7.6 (5.7) SD (+ 2.3) Pneumonia (2)
19 3 years/F CCLA Thoracic and mediastinal Acute pancreatitis, chylothorax, and coagulation disorder Steroids and octreotide 14 7.8–14.3 (10.3) SD (−0.1) Hyperlipidemia (2)
20 55 years/F CCLA Abdominal cavity and intestinal tract Anemia, intestinal lymphangiectasia, and protein losing enteropathy Transfusion, octreotide, albumin, 6 (death from progression of disease) 2.4–7.7 (4.8) SD (−2.0) Stomatitis (1)
  1. M male, F female, LM lymphatic malformation, KLA kaposiform lymphangiomatosis, GLA generalized lymphatic anomaly, CCLA central conducting lymphatic anomaly, PR partial response, SD stable disease, CTCAE Common Terminology Criteria for Adverse Events