PMID- 15820939
OWN - NLM
STAT- MEDLINE
DCOM- 20060525
LR  - 20220330
IS  - 1592-8721 (Electronic)
IS  - 0390-6078 (Linking)
VI  - 90
IP  - 4
DP  - 2005 Apr
TI  - Efficacy and safety of sildenafil in the treatment of severe pulmonary 
      hypertension in patients with hemoglobinopathies.
PG  - 452-8
AB  - BACKGROUND AND OBJECTIVES: During the last decade new approaches to the treatment 
      of pulmonary arterial hypertension (PH) have increased symptomatic relief and 
      prolonged survival. PH is a common sequel of the hemoglobinopathies, thalassemia 
      and sickle cell anemia, but the use of standard oral treatment options, such as 
      calcium channel blockers, endothelin receptor antagonists, and long-term 
      anticoagulation therapy, is limited because of toxicity and poor effectiveness. 
      Sildenafil citrate is a selective and potent inhibitor of cGMP-specific 
      phosphodiesterase-5 (PDE5) which promotes selective smooth muscle relaxation in 
      lung vasculature and has been utilized successfully in the treatment of PH. The 
      primary objective of this study was to evaluate the efficacy of sildenafil 
      treatment in the control of PH in patients with hemoglobinopathies. DESIGN AND 
      METHODS: In this study patients with hemoglobinopathies (thalassemia intermedia 
      n=4; thalassemia major n=2; sickle thalassemia n=1) suffering from severe PH were 
      treated with sildenafil citrate (50 mg b.i.d.) for periods ranging from 4 to 48 
      months. RESULTS: A significant decrease in pulmonary pressure and improvement in 
      exercise capacity and functional class were observed in all patients. No 
      significant adverse events were reported. INTERPRETATION AND CONCLUSIONS: These 
      data, in a small group of patients, indicate that sildenafil citrate is effective 
      in the treatment of PH in hemoglobinopathies that cannot be treated with 
      alternative oral drugs and is well tolerated long-term at a daily dose of 100 mg, 
      though studies including more patients may uncover toxicities and limitations of 
      efficacy.
FAU - Derchi, Giorgio
AU  - Derchi G
AD  - Struttura Complessa di Cardiologia, Ospedali Galliera, Genoa, Italy. 
      giorgio.egildo.derchi@galliera.it
FAU - Forni, Gian Luca
AU  - Forni GL
FAU - Formisano, Francesco
AU  - Formisano F
FAU - Cappellini, Maria Domenica
AU  - Cappellini MD
FAU - Galanello, Renzo
AU  - Galanello R
FAU - D'Ascola, Giandomenico
AU  - D'Ascola G
FAU - Bina, Patrizio
AU  - Bina P
FAU - Magnano, Carmelo
AU  - Magnano C
FAU - Lamagna, Martina
AU  - Lamagna M
LA  - eng
PT  - Journal Article
PT  - Multicenter Study
PL  - Italy
TA  - Haematologica
JT  - Haematologica
JID - 0417435
RN  - 0 (Piperazines)
RN  - 0 (Purines)
RN  - 0 (Sulfones)
RN  - 0 (Vasodilator Agents)
RN  - BW9B0ZE037 (Sildenafil Citrate)
SB  - IM
CIN - Haematologica. 2005 Apr;90(4):433-4. PMID: 15820926
CIN - Haematologica. 2005 Apr;90(4):441-4. PMID: 15820937
MH  - Administration, Oral
MH  - Adult
MH  - Blood Transfusion
MH  - Clinical Trials as Topic
MH  - Dose-Response Relationship, Drug
MH  - Echocardiography, Doppler
MH  - Female
MH  - Hemoglobinopathies/*complications
MH  - Humans
MH  - Hypertension, Pulmonary/complications/*drug therapy
MH  - Male
MH  - Piperazines/adverse effects/*therapeutic use
MH  - Priapism/chemically induced/drug therapy
MH  - Purines
MH  - Severity of Illness Index
MH  - Sildenafil Citrate
MH  - Sulfones
MH  - Vasodilator Agents/adverse effects/*therapeutic use
EDAT- 2005/04/12 09:00
MHDA- 2006/05/26 09:00
CRDT- 2005/04/12 09:00
PHST- 2005/04/12 09:00 [pubmed]
PHST- 2006/05/26 09:00 [medline]
PHST- 2005/04/12 09:00 [entrez]
PST - ppublish
SO  - Haematologica. 2005 Apr;90(4):452-8.