Table 2.
The summary of patient characteristics and treatment response.
| Patient number | Diagnose | Age at initiation of sirolimus(years) | sex | Location of lesions | Previous treatment (duration) / Disease response of previous treatment | Duration of sirolimus(years) | Additional treatment with sirolimus | Target mass size change (% of compared to the previous lesion size) | Self-reported changes in symptoms | Disease Response (Ref 8 / Ref 7) | Adverse Effects (CTCAE Grade) | Current status |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | KHE | 0.2 | F | Lt arm | Pred (3wk), PPL (2wk) / Poor | 3.5 | N/A | 8.6 × 4.3 × 6.8 cm → 2.2 × 2.0 × 6.9 cm (12.1%) | The swelling, redness and heat sensation on the lesion improved. | PR/ Intermediate | Pneumocystis Jiroveci infection (3) | Reinitiation after D/Ca, Ongoing |
| 2 | LM | 6.1 | F | Lung, pericardium, abdomen, spleen | IVIG #1, Pred,(7mo) Embolization #2 / Poor | 3.4 | N/A | Unmeasurable due to extensive and diffuse lesions | No reported specific change. | SD/ Poor | dyspepsia (1), fatigue (2) | Reinitiation after D/Cb, Ongoing |
| 3 | LM | 0.1 | M | Neck | None | 3.3 | Sclerotherapy #1 | 4.5 × 3.0 × 2.7 cm → 6.2 × 1.9 × 3.2 cm (103.4%) | Initial respiratory difficulty improved; otherwise, stationary. | SD/ Poor | recurrent upper respiratory infection (2) | D/C |
| 4 | LM | 1.1 | M | Forehead, orbit | None | 3.3 | Sclerotherapy #3 | 1.4 × 4.9 × 3.3 cm → 0.8 × 4.5 × 2.8 cm (44.5%) | Right eye opened wider. | PR/ Intermediate | none | ongoing |
| 5 | LM | 2.4 | F | Neck | Sclerotherapy #3 / Poor | 2.8 | Excision of lymphangioma #1 | 5.8 × 5.5 × 3.7 cm → 2.7 × 5.0 × 3.1 cm (35.5%) | No reported specific change. | PR/ Intermediate | recurrent upper respiratory infection (2) | Reinitiation after D/Cc Ongoing |
| 6 | LM | 16.1 | F | Buttock | Mass excision #1 / Intermediate | 2.7 | N/A | 9.2 × 4.4 × 10.0 cm → 8.6 × 3.3 × 9.2 cm (64.5%) | Buttock pain improved | PR/ Intermediate | none | ongoing |
| 7 | LM | 11.6 | M | Buttock, thigh, perianal area | mass excision #2, Electrocauterization #2 / Intermediate | 0.6 | N/A | Unmeasurable due to extensive and diffuse lesions | No reported specific change. | SD/ Poor | Hyperglycemia (1) | D/C |
| 8 | KHE | 0.2 | M | Rt cheek | Pred (1 mo), PPL (1 mo) / Poor | 2.2 | Pred (6m), VCR (2m) | 6.1 × 4.0 × 4.4 cm → 3.3 × 2.3 × 2.4 cm (17.0%) | The swelling, redness and heat sensation on the lesion improved. | PR/ Intermediate | hypercholesterolemia (1) | D/C |
| 9 | LM | 13.8 | M | Neck, tongue | Excision#2, Sclerotherapy #2, glossoplasty #1 / Poor | 1.1 | N/A | Unmeasurable due to diffuse lesions | Tongue discomfort and bleeding did not change. | SD/ Poor | oral mucositis (2) | D/C |
| 10 | KHE | 1.1 | M | scapula and acromion | Pred (4 mo) / Intermediate | 4.3 | Pred (5mo) | 2.5 × 1.0 × 2.3 cm → 1.9 × 0.9 × 1.4 cm → none | CR/ Good | herpatic gingivostomatitis (2) | D/C | |
| 11 | CH | 0.4 | M | Rt. Back, flank | Pred (2 mo), PPL (2 mo) / Poor | 1 | N/A | 6.0 × 5.0 cm → 5.0 × 5.0 cmd | The swelling, redness and heat sensation on the lesion improved. | PR/ Intermediate | none | ongoing |
| 12 | VM | 2 | F | Rt. Check, lip | Laser treatment, topical PPL / Poor | 1 | N/A | Unmeasurable due to diffuse lesions | No reported specific change. | SD/ Poor | Oral mucositis (2) | D/C |
| 13 | KHE | 4 | M | cheek mass | Pred (1 mo), PPL (1 mo) / Intermediate | 2.1 | N/A | 2.7 × 1.7 × 2.0 cm → 2.3 × 1.2 × 1.9 cm (57.1%) | The swelling, redness and heat sensation on the lesion improved. | PR/ Intermediate | none | ongoing |
| 14 | VM | 18.8 | F | Rt. neck, mandible, mouth floor, oropharynx | None | 1 | N/A | Unmeasurable due to extensive and diffuse lesions | Oral pain improved | PR/ Intermediate | oral mucositis (2) | ongoing |
| 15 | KHE | 12.8 | M | Thigh, Foot | Curettage #1 / Poor. | 1.1 | N/A | 6.0 × 3.0 × 10.0 → 3.9 × 3.6 × 8.8 cm (68.7%) | Pain improved | PR/ Intermediate | none | ongoing |
| 16 | KHE | 0.2 | M | neck mass | Pred (4 mo), PPL (3 mo) / Poor | 1.4 | N/A | 6.0 × 3.6 × 2.8 cm → 4.0 × 3.2 × 2.7 cm (57.1%) | No reported specific change. | PR/ Intermediate | none | ongoing |
| 17 | VM | 9.9 | M | Rt. Leg, Hip, Foot | None | 2.1 | Sclerotherapy #4 | Unmeasurable due to extensive and diffuse lesions | The overall pain and heaviness in the right leg have improved. | PR/ Intermediate | none | ongoing |
| 18 | GSD | 14 | M | Pelvic cavity, Abdomen, Rt. Leg to Axilla | Thoracic duct embolization #1 / Poor | 3.6 | Pamidronate, Lymphovenous anastomosis, radiotherapy | Unmeasurable due to extensive and diffuse lesions | Pleural effusion and lymphedema waxed and waned. | SD/ Poor | none | ongoing |
| 19 | GSD | 8.6 | M | Rt iliac bone, lumbosacral vertebral bodies, inguinal | None | 1 | N/A | Unmeasurable due to extensive and diffuse lesions | Pelvic pain waxed and waned | SD/ Poor | non-cardiac chest pain (1), sinus tachycardia (1) | ongoing |
| 20 | VM | 14.5 | M | Chest, shoulder | Laser treatment, topical PPL / Poor | 0.6 | N/A | Unmeasurable due to diffuse skin lesions | The overall red color has lightened, but the extent of the lesion remains similar | SD/ Poor | oral mucositis (1) | ongoing |
CH, Congenital Hemangioma; CR, Complete Response; GSD, Gorham-Stout Disease; D/C, discontinued; IVIG, Intravenous immunoglobulin; KHE, Kaposiform Hemangioendothelioma; LM, Lymphatic Malformation; N/A, not applicable; PD, Progressive Disease; PR, Partial Response; Pred, Prednisolone; PPL, propranolol; SD, Stable Disease; VCR, Vincristine; VM, Venous Malformation.
a
Restarted sirolimus after a 9-month period off due to an increase in the size of the lesion.
b
Restarted sirolimus after 6-month period off due to relapse of pleural effusion.
c
Restarted sirolimus after 3-month period off due to an increase in the size of the lesion.
d
Evaluated by physical exam. Depth could not be measured, however the previously swollen lesion has significantly flattened.