Table 1.
Summary of KLA patients
| N | Age at start /sex | Location of target lesions | Major signs, symptoms, and/or complications | Previous treatments | Duration of sirolimus treatment (months) | Range (mean) of trough concentrations (ng/mL) | Response to sirolimus |
|---|---|---|---|---|---|---|---|
| 1a | 2 years/M | Mediastinum, abdominal cavity, pelvic cavity, bilateral inguinal regions and testes | Abdominal distention, pain and coagulation disorder | Corticosteroid, immune globulin and platelet transfusion | 6 | 7.8–12.7 (10.4) | PD |
| 2a | 4 months/F | Left mediastinum and left lung | Dyspnea pleural effusion and coagulation disorder | Thoracic drainage | 18 | 6.1–15.7 (10.2) | PR |
| 3a | 5 years/M | Lungs, vertebrae, ribs and mediastinum | Pleural effusion and coagulation disorder | Thoracic drainage, fibrinogen transfusion, exploratory thoracotomy | 42 | 10.7–14.9 (13.3) | PR |
| 4a | 6 months/M | Lungs, mediastinum, spleen, liver, pancreas and mesentery | Abdominal distention, vomiting and coagulation disorder | Vincristine plus corticosteroid, aspirin | 41 | 5.1–13.7 (11.6) | SD |
| 5a | 14 months/M | Lungs and spleen | Cough, respiratory distress, pleural effusion, pericardial effusion and coagulation disorder | Pericardiocentesis, corticosteroid, splenectomy and platelet transfusion | 14 | 5.9–14.3 (12.1) | PR |
| 6a | 4 years/M | Left lung and mediastinum | Cough and coagulation disorder | Sirolimus, sirolimus plus steroids, sirolimus and vincristine | 21 | 9.4–15.8 (10.6) | SD |
| 7a | 2 years/M | Mediastinum, lungs, neck and spleen | Dyspnea, cough and coagulation disorder | Propranolol and prednisone | 21 | 10.0–14.7 (13.2) | SD |
| 8b | 8 years/M | Bone, thoracic and mediastinal | Chylothorax and coagulation disorder | Interferon and propranolol | 30 (cessation) | 4.4–9.0 (7.5) | PR |
| 9b | 8 years/M | Bone, thoracic and mediastinal | Scoliosis, chylothorax and coagulation disorder | Steroids and propranolol | 24 | 8.1–12.4 (11.2) | SD |
| 10b | 20 years/M | Bone, thoracic and right chest wall | Gastrointestinal hemorrhage and coagulation disorder | Steroids | 12 | 4.7–6.0 (5.5) | PR |
| 11b | –/M | Thorax | NG | NG | NG | NG | PD |
| 12b | 7 years/M | Left scapular, left arm and thoracic vertebral bodies | Dyspnea, shortness of breath, pleural effusion and coagulation disorder | Chinese traditional medicine, pleurocentesis and albumin | 8 | 9.3–13.4 (12.3) | PR |
| 13b | NG | NG | NG | NG | 12 | 10.0–15.0 (NG) | PR |
| 14b | NG | NG | NG | NG | 12 | 10.0–15.0 (NG) | PR |
| 15b | NG | NG | NG | NG | 12 | 10.0–15.0 (NG) | PR |
| 16b | NG | NG | NG | NG | 12 | 10.0–15.0 (NG) | PR |
| 17b | NG | NG | NG | NG | 12 | 10.0–15.0 (NG) | PR |
| 18b | NG | NG | NG | NG | 12 | 10.0–15.0 (NG) | PR |
| 19b | NG | NG | NG | NG | 12 | 10.0–15.0 (NG) | PD |
| 20b | 7 years/M | Abdominal cavity, lower neck, mediastinum, bone and left upper arm | Cephalohematoma and coagulation disorder | Steroids, vincristine and zoledronate | 12 | 9.4–14.8 (NG) | PR |
| 21b | 13 years/F | Bone, mediastinum, spleen | Cough, fever, fatigue, intermittent wheezing, pleural effusion and coagulation disorder | Thoracostomy | 3 | NG | PR |
| 22b | 20 months/M | Mediastinum and spleen | Cough, pleural effusion, pericardial effusion, epidural hematoma and coagulation disorder | Diaphragmatic fenestrations | 7 | NG | PD |
| 23b | 11 years/F | Mediastinum, bone and spleen | Cough, chest pain, fatigue, listlessness, weight loss, coagulation disorder and pericardial effusion, coagulation disorder | Pericardiocentesis | 31 | NG | SD |
| 24b | 4 years/M | Bone, spleen and mediastinum | Fatigue, decreased appetite, weight loss, cough, spontaneous bruising, back pain, coagulation disorder and pleural effusion | Cryoprecipitate, platelet infusions and prednisolone | NG | NG | SD |
M male, F female, NG not given, PD progressive disease, PR partial response, SD stable disease
aData from our center
bData from literature