Skip to main content

Table 1 New treatments in HHT, case reports and case series

From: Future treatments for hereditary hemorrhagic telangiectasia

Ref.

Treatment

n

(HHT Type)

Sex/ Age

Symptoms

Treatment indication

Treatment

Efficacy of treatment

[44]

Tacrolimus

1

(HHT2)

M 51

Epistaxis

GI bleeding

Epistaxis

Dose unknown

↓ Epistaxis

[45]

Pazopanib

1

(HHT2)

M 61

Epistaxis

Anemia

Epistaxis

50 mg/d during 1 month

then: 100 mg/d

↓Epistaxis

[46]

Pazopanib

7

(3 HHT1, 3 HHT2, 1 JP/HHT)

 

Anemia

Epistaxis

Anemia, OR, severe epistaxis with iron deficiency

50 mg /d during 12 weeks

↓epistaxis duration

Hb

SF-36

[47]

Nintedanib

1

(HHT2)

M 70

Epistaxis

Telangiectasias

Pulmonary fibrosis

300 mg/d

↓Epistaxis and telangiectasias

[48]

Sunitinib

1

(?)

M 68

Epistaxis

Multiple metastases

Oncology

37.5 mg/d

↓ epistaxis frequency and intensity

↓facial telangiectasia

[49]

Buparlisib

1

(HHT2)

F 49

Epistaxis

Oncology

100 mg/d

↓ frequency of epistaxis