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Table 1 Published studies of therapies in ES (from [39])

From: Strategies for the management of pulmonary arterial hypertension in patients with congenital heart disease

Class

First author

Year

Drug

N

FC % change II/III/IV

or mean

RCT

Main findings

ERA

Galiè [20]

2006

Bosentan

54

0/100/0

Yes

↓PVRi, ↓mPAP,↑6MWD

 

Zuckerman [40]

2011

Ambrisentan

17

NR

No

↑6MWD

 

Blok [28]

2017

Macitentan

40

52/48/0

No

↓FC, ↓NT-proBNP, ↔6MWD

 

Herbert [29]

2017

Macitentan

15

NR

No

↑6MWD, ↑SaO2

 

Galie [41]

2017

Macitentan

226

60/40/0

Yes

↔6MWD, ↓NT-proBNP, ↓PVRi

PDE5i

Mukhopadhyay [7]

2006

Sildenafil

16

69/31/0

No

↑6MWD, ↑SaO2, ↓mPAP, ↓PVR

 

Chau [42]

2007

Sildenafil

7

3.3 ± 0.7

No

↓FC, ↑SaO2, ↓mPAP, ↓PVR

 

Garg [10]

2007

Sildenafil

21

46/54/0

No

↓FC, ↑6MWD, ↑SaO2, ↓mPAP, ↓PVRi

 

Tay [11]

2011

Sildenafil

12

0/100/0

No

↓FC, ↑6MWD, ↑HRQOL

 

Zhang [12]

2011

Sildenafil

84

52/39/8

No

↑6MWD, ↑SaO2, ↓mPAP, ↓PVRi

 

Mukhopadhyay [14]

2011

Tadalafil

28

79/21/0

Yes

↓FC, ↑6MWD, ↓PVR

 

Sun [13]

2013

Sildenafil

29

NR

No

↓FC, ↑6MWD, ↓PVR

Prostanoid

Rosenzweig [32]

1999

Epoprostenol (intravenous)

20

15/50/35

No

↓mPAP, ↑CI, ↓PVRi,↑SvO2

 

Fernandes [33]

2003

Epoprostenol (intravenous)

8

0/25/75

No

↑6MWD

 

Thomas [43]

2013

Epoprostenol (intravenous) & Treprostinil (subcutaneous)

9

12/88/0

No

↓mPAP, ↑SvO2

 

Skoro-Sajer [44]

2018

Treprostinil (subcutaneous)

22

0/47/53

No

↓FC, ↓PVR, ↓NT-proBNP, ↑6MWD

 

Yang [36]

2012

Iloprost (nebulised)

12

0/75/25

No

↓FC, ↑6MWD, ↑SaO2,

 

Cha [37]

2013

Iloprost (nebulised)

13

0/69/31

No

↓FC, ↑6MWD, ↑HRQoL

 

Chon [38]

2017

Iloprost (nebulised)

11

0/64/36

No

↓FC, ↓mPAP, ↓PVR, ↑SaO2, ↑6MWD

Combination

Iversen [45]

2010

Bosentan +/− Sildenafil

21

43/48/5

Yes

↔6MWD, ↑SaO2

 

D’Alto [46]

2012

Bosentan & Sildenafil

32

2.9 ± 0.3

No

↓FC,↑6MWD, ↓mPAP, ↓PVR, ↓NT-proBNP

 

Sitbon [30]

2020

Macitentan & Tadalafil

46

63% improved FC

No

↓PVR, ↓NT-proBNP

  1. Abbreviations and legend. CI: Cardiac index, FC: Functional class, HRQoL: Health related quality of life, mPAP: mean pulmonary artery pressure, NT-proBNP: N terminal pro-Brain natriuretic peptide, PVR(i): Pulmonary vascular resistance (indexed), SaO2: peripheral oxygen saturation, 6MWD: 6 min walk distance, SvO2: mixed venous oxygen saturation ↑: Increased, ↓:Decreased, ↔: No change
  2. Reproduced and updated from Condliffe R, Clift P, Dimopoulos K, Tulloh RMR. Management dilemmas in pulmonary arterial hypertension associated with congenital heart disease. Pulmonary Circulation 8(3), pp. 1–12. Copyright© 2018 by the Authors. Reprinted by permission of SAGE Publications, Ltd.