Table 5

Therapeutic trials for gastrointestinal (GI) bleeding in hereditary haemorrhagic telangiectasia (HHT)

StudySubjectsNo with HHTMean (range) age (years)Intervention% with follow-upMean follow-up (months)Post-treatment outcomeFrequency post-treatment outcomeProcedural complicationFrequency complication
Bown et al 1985,109 Case series18, severe GI bleeding, transfusion dependent8/18 (44%)62 (42–74)
  • 100%

  • 2 APC

  • 6 ND-YAG (mean 7 sessions)

100%14
  • Reduced transfusions

  • No further transfusions

  • Recurrence requiring surgery

  • 8/8 (100%)

  • 3/8 (38%)

  • 3/8 (38%)

Perforation0%
Gostout et al 1988,111 Case series93, severe GI bleeding, transfusion dependent10/93 (11%)63100% ND-YAG (2–6 sessions)100%15Reduced transfusions9/10 (90%)
  • Perforation

  • Delayed bleeding

  • 3/93 (3%)

  • 5/93 (5%)

Sargeant et al 1993,110 Case series41, severe GI bleeding, transfusion dependent9/41 (22%)66 (55–81)100% ND-YAG (repeated sessions)100%51
  • Reduced or stabilised number of transfusions

  • Reduction in mean yearly transfusions pre–post

  • 6/9 (67%)

  • 8 (4–42) vs 4 (0–44)

  • Perforation

  • Antral narrowing

  • 1/41 (2%)

  • 2/41 (4%)

Van Cutsem et al 1990,106 Placebo-controlled cross-over trial10, severe GI bleeding from VMs, transfusion dependent6/10 (60%)65–89100% ethinylestradiol+norethisterone versus placebo100%6
  • Reduced mean transfusions pre–post*

  • No further bleeding

  • p<0.002

  • 5/6

  • Death (MI)

  • Feminising

  • Vaginal bleeding

  • 1/10 (10%)

  • 1/10 (10%)

  • 2/10 (20%)

Longacre et al 2003,103 Case series43, HHT-related GI bleeding43/43 (100%)57 (33–78)
  • 23/43 (53%)

  • Medical therapy

  • 19 ethinylestradiol/norethindrone

  • 2 danacrine

  • 2 aminocaproic acid

100%18Mean haemoglobin pre–post
  • 8.6 vs 9.9

  • p=0.0018

DVT1/19 (5%)
  • * Mean for all 10 patients (HHT and non-HHT).

  • APC, argon plasma coagulation; DVT, deep vein thrombosis; MI, myocardial infarction; Nd-YAG, neodymium-doped yttrium aluminium garnet laser.