Table 2.
Characteristics of patients treated with sirolimus
| 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) |
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