NATIONAL INSTITUTE FOR HEALTH AND CLINICAL EXCELLENCE

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1 NATIONAL INSTITUTE FOR HEALTH AND CLINICAL EXCELLENCE INTERVENTIONAL PROCEDURES PROGRAMME Interventional procedure overview of percutaneous fetal balloon valvuloplasty for aortic stenosis 322 Introduction This overview has been prepared to assist members of the Interventional Procedures Advisory Committee (IPAC) in making recommendations about the safety and efficacy of an interventional procedure. It is based on a rapid review of the medical literature and specialist opinion. It should not be regarded as a definitive assessment of the procedure. Date prepared This overview was prepared in September Procedure name Percutaneous fetal aortic balloon valvuloplasty Specialty societies Paediatric Intensive Care Society British Paediatric Cardiac Association Royal College of Obstetricians and Gynaecologists British Maternal and Foetal Medicine Society Description Description Congenital heart defects are the most common type of birth defects and include aortic valve stenosis (narrowing), the severity of aortic valve stenosis ranges from mild to severe, severe cases are rare but carry a high rate of postnatal mortality. Severe aortic stenosis when present early in fetal life results in left ventricular dysfunction, which may initially produce left ventricular dilation however as the myocardium becomes damaged there is an arrest of left ventricular growth which can lead to hypoplastic left heart syndrome (HLHS) 1. Obstruction to the left ventricular outflow tract leads to increased pressure in the left ventricle and atrium. If the foramen ovale is patent blood flow passes from left to right however if the foramen ovale closes before birth the resulting high left sided pressure produces fibrosis of the myocardium and pulmonary venous hypertension with arterialisation of the pulmonary veins. This condition is known as aortic stenosis with a restrictive interatrial communication has a very poor prognosis. In addition the mitral valve and IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 1 of 19

2 aortic arch may be underdeveloped in HLHS, if severe the left ventricular outflow tract is unable to sustain systematic circulation. Diagnosis of is often made at the routine screening ultrasound performed at about 20 weeks of pregnancy. Many fetuses diagnosed with severe aortic stenosis will survive until birth. However about 10% will die in utero from either hydrops associated with a restrictive interatrial communication or from aneuploidy. This along with a mortality rate of approximately 50% during the first year of life leads some parents to choose termination of pregnancy. Prognosis is better for those babies in whom a biventricular repair is possible, but for those with only a univenticular heart the morphological right ventricle tends to fail during the teenage years; these children are candidates for cardiac transplant however this is not straight forward due to their previous complex surgery. If the pulmonary vascular bed has been damaged by restrictive interatrial communication the resulting pulmonary hypertension can prove fatal during infancy. Current treatment and alternatives The majority of congenital heart defects can be treated after birth with good outcomes and in these cases in-utero intervention would be unnecessary. For other defects staged surgical palliation is the only option 1. Immediately after birth a prostaglandin infusion is started to keep the Ductus Arteriosus open and investigation undertaken to establish the severity of HLHS. For babies born with an adequate biventricular heart and aortic valve disease, postnatal balloon valvuloplasty is the initial preferred option to encourage remodelling and growth of the left ventricle. Further balloon valvuloplasty is often required with later valve replacement. Babies born with HLHS are assessed for the suitability for staged reconstructive surgery. The option of heart transplantation is not currently a reality for most babies delivered in the UK. Staged reconstruction for HLHS requires up to three operations over 3 or more years and involves complex high risk open-heart surgery. The first is the Norwood procedure is usually performed in the first week or two of life while the Ductus Arteriosus is still open and involves attaching the pulmonary artery to the aorta, inserting a Gore-Tex shunt between the pulmonary artery and the right ventricle or the aorta and finally an atrial septectomy. This long complex open heart operation carries a high risk of intraoperative and post operative mortality. A number of babies die within the first few days or months or life despite successful surgery due to the extent of secondary aortic and myocardial disease or from persistent pulmonary hypertension. As the child grows a more permanent blood supply to the lungs is required. Between 3 to 9 months of age either a Cavo Pulmonary Shunt (Glenn Shunt) or a Hemi- Fontan procedure is performed. This surgery carries fewer risks than the first stage operation and involves connecting the Superior Vena Cava to the pulmonary artery and removal of the Gore-Tex shunt. The final surgical operation the Fontan procedure is performed when the child starts to shows symptoms of increased breathlessness and slowing growth which in some occurs before school age. Here using one of two methods (baffle or Gore-Tex conduit) the inferior vena cava is connected to the pulmonary artery thereby ensuring all blood returning from the body flows directly to the lungs. IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 2 of 19

3 What the procedure involves: The aim of the fetal aortic valvuloplasty is to reduce valvular stenosis thereby halting in-utero damage to the ventricular muscle and preventing the progression of pulmonary vascular bed damaged coursed by restrictive interatrial communication, in the hope of allowing greater success with surgery in the period after birth with preservation of a biventricular heart. Fetal aortic valvuloplasty may be considered where there is an expected high postnatal mortality or morbidity and progressively worsening disease in the fetus. Improvements in fetal imaging have assisted with this identification. The procedure is performed at weeks gestation under local anaesthesia (with maternal sedation) and using ultrasound guidance. A cannula and stylet needle are advanced through the maternal abdomen, uterine wall and fetal chest wall into the left ventricle of the fetus. A guidewire is then inserted through the needle and across the aortic valve. A balloon catheter is then inserted and inflated across the stenotic valve. The balloon catheter and needle are then withdrawn. Fetal positioning is critical in determining the success of the procedure. Efficacy: Fetus There is limited published data on this procedure. The largest published series reports on 20 fetuses that underwent in utero aortic valvuloplasty. Technical success was achieved in 70% (14/20) with a significant difference demonstrated in the growth of the mitral valve, aortic valve and ascending aorta compared to those that declined the procedure or were technically unsuccessful (n=10). Of the 14 technical successes, 3 were born with a biventricular heart, 6 were born with HLHS, 2 died in utero and a further 3 were unborn at the time of writing. In a second series of 12 fetuses with severe obstructions of the aortic valve, there were 7 technical successes with only one alive at the time of writing. Mother Few studies reported on maternal outcomes. In one study, maternal hospital stay was 2 days (range 1-7 days). Specialist Advisors The Specialist Advisors noted the difficulty in determining appropriate selection of cases and whether any benefits accruing from in utero intervention outweighed the risks of the procedure. The lack of long-term data was also noted. Safety Fetus There were 3 deaths from the procedure in a study of 20 fetuses. Two fetuses died within 24 hours of having the procedure, one due to severe hydrops and the other a result of fetal stress. The third fetus died three days after the procedure due to severe bradycardia. In a second study of 12 fetuses, four died within 24 hours of the procedure. Causes of death were persistent bradycardia (n=2), bleeding (n=1) and one at valvotomy after emergency delivery. Both studies listed balloon rupture and intraoperative fetal bradycardia as further complications. In the study of 20 fetuses, bradycardia occurred in 15 cases, with medication required in 73% (11/15). IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 3 of 19

4 Mother Maternal morbidity was primarily related to the need for emergency caesarean following fetal valvuloplasty. Three emergency caesareans were undertaken in a study of 12 fetuses, two for sustained bradycardias and one for chorioamnionitis 322 Specialist Advisors The Specialist Advisors listed fetal death, bleeding, bradycardia, pericardial effusion and balloon rupture as potential complications. They also noted that there was a risk of premature labour and possible maternal morbidity associated with the use of anaesthesia. Literature review Rapid review of literature The medical literature was searched to identify studies and reviews relevant to percutaneous fetal aortic valvuloplasty. Searches were conducted via the following databases, covering the period from their commencement to September 2005: Medline, PreMedline, EMBASE, Cochrane Library and other databases. Trial registries and the Internet were also searched. No language restriction was applied to the searches. (See Appendix C for details of search strategy.) The following selection criteria (Table 1) were applied to the abstracts identified by the literature search. Where these criteria could not be determined from the abstracts the full paper was retrieved IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 4 of 19

5 Table 1 Inclusion criteria for identification of relevant studies Characteristic Publication type Patient Intervention/test Outcome Language Criteria Clinical studies were included. Emphasis was placed on identifying good quality studies. Abstracts were excluded where no clinical outcomes were reported, or where the paper was a review, editorial, laboratory or animal study. Conference abstracts were excluded unless they were thought to add substantively to the published evidence base Fetuses with aortic stenosis or atresia Inutero balloon valvuloplasty Articles were retrieved if the abstract contained information relevant to the safety and/or efficacy. Non-English-language articles were excluded unless they were thought to add substantively to the English-language evidence base. List of studies included in the overview There were few published studies on this procedure. This overview was based on five published papers and two abstracts. Two of the published papers were case reports 2 3, with both cases included in a more recent published paper 6. No other clinical studies were identified. Existing reviews on this procedure There were no published reviews identified at the time of the literature search. Related NICE Guidance: Below is a list of NICE guidance related to this procedure. Appendix B details the recommendations made in each piece of guidance listed below. Interventional Procedures: Published: IPG 078 Balloon valvuloplasty for aortic valve stenosis IPG067 IPG075 IPG076 IPG 077 IPG 095 Balloon dilatation of pulmonary valve stenosis Balloon angioplasty of pulmonary vein stenosis in infancy Balloon dilatation with or without stenting for pulmonary artery or non-valvular right ventricular outflow tract obstruction in children Balloon dilation of systemic to pulmonary arterial shunts in children Radiofrequency valvotomy for pulmonary atresia In development: 323 Fetal aortic valvuloplasty Technology Appraisals: None relevant Guideline Development None relevant Public Health None relevant IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 5 of 19

6 Table 2 Summary of key efficacy and safety findings on percutaneous fetal aortic balloon valvuloplasty Abbreviations used: AS aortic stenosis, LV left ventricle, HLHS hypoplastic left heart syndrome, PAIVS pulmonary atresia with intact ventricular septum Study Details Key efficacy and safety findings Key efficacy and safety findings Comments Tworetzky et al (2004) 4 US January Case series 24 fetuses (only 20 reported on) - 22 with aortic stenosis - 2 with aortic atresia Gestational age: weeks. Selection criteria: Fetuses with a gestational age under 29 weeks, aortic stenosis or atresia as the dominant lesion, favourable anatomy i.e. salvageable left heart, and a progressively worsening disease. Disclosure of interest: study was funded by the Burnes Family Research Endowment and the Marram and Carpenter Fund Outcomes measured: left ventricle function, growth of the left heart structures and colour Doppler flow patterns. Twenty fetuses underwent AS dilatation, with technical success in 14. Outcomes of 14 technical success - 3 live born with biventricular heart - 6 live born with HLHS - 2 died in utero - 3 still awaiting birth Outcomes of 6 technical failures - 3 live born with HLHS - 1 termination - 2 premature or died in utero demise From the graphs provided, the authors note that there was significant growth of the mitral valve, aortic valve and ascending aorta and a non-significant trend towards LV growth in fetuses with successful valvotomies (n=12) compared with unsuccessful and control cases (n=10) (no absolute numbers given). At delivery all infants were assessed for HLHS. All 3 surviving infants with failed in utero procedures and 6 of 9 survivors with successful procedures had evidence of HLHS. Complications related to procedure 3 deaths - one fetus died one day after the procedure due to severe hydrops - one fetus died one day after the procedure thought to be related to fetal stress - one fetus died three days after the procedure related to severe bradycardia Other complications: - one previable delivery due to an incompetent cervix 3 weeks after the procedure - balloon rupture. In one case a balloon fragment was missing Intraoperative fetal bradycardia occurred in 15 cases requiring medication in 11 cases - 2 fetuses had a small pericardial effusion that resolved spontaneously Maternal - one woman required oxygen administration. Author report that none of the mothers experienced haemorrhage requiring blood transfusion, postoperative infection or thrombotic events. From the original study population of 24 fetuses, 4 sets of parents declined further intervention 3 were carried to term delivery (all had HLHS) and one was terminated. After a high rate of technical failure in the first 4 patients the technique was modified. Fetuses with technically successful procedures were analysed separately from those who declined or failed the procedure. Authors note that timing of invention seems important. IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 6 of 19

7 Abbreviations used: AS aortic stenosis, LV left ventricle, HLHS hypoplastic left heart syndrome, PAIVS pulmonary atresia with intact ventricular septum Study Details Key efficacy and safety findings Key efficacy and safety findings Comments Kohl et al (2000) 6 Case series cases from six international centres including Guy s and St Thomas Hospital UK fetuses with severe obstructions of the aortic valve - 8 severe aortic stenosis - 2 aortic valve atresia - 2 severe aortic valve stenosis associated with PAIVS. Mean gestational age at detection was 25.7 weeks (range weeks) Mean gestational age at intervention was 29.2 weeks (range weeks) Selection criteria: observation of progressive disease and/or the expected poor outcomes such as hydrops in the setting of heart disease. Disclosure of interest: not stated There were 7 technically successful balloon valvuloplasties; none of the fetuses had an atretic valve. Only one fetus (1/7) survived 2. Of the 5 technical failures, 1 fetus with severe aortic valve stenosis underwent successful postnatal intervention and was alive at time of writing. Six fetuses who survived prenatal intervention regardless of technical success died from cardiac dysfunction or at surgery in the first days or weeks after delivery. 4 died early within 24 hours. Maternal hospital stay was 2 days (range 1-7 days) Four fetuses died within 24 hours after percutaneous valvuloplasty - 1 from bleeding complication - 2 from persistent bradycardia - 1 at valvotomy after emergency delivery Technical failures - 6 cases of balloon rupture - 8 fetuses experienced bradycardia (either intermittently or sustained) Maternal morbidity was related to the need for emergency caesarean in 2 cases for sustained bradycardia and one case of chorioamnionitis. Procedure was called technically successful if the balloon catheter could be inflated across the obstructed valve and echocardiograph evidence of relief of valvular obstruction was found after the procedure. Included reports from 2. Authors noted that satisfactory alignment could not be achieved in about half of the fetuses in the study, in all of whom valvular dilatation failed. Repeated cardiac punctures to improve needle alignment may result in fetal bleeding and death due to injury to cardiac structures.. IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 7 of 19

8 Abbreviations used: AS aortic stenosis, LV left ventricle, HLHS hypoplastic left heart syndrome, PAIVS pulmonary atresia with intact ventricular septum Study Details Key efficacy and safety findings Key efficacy and safety findings Comments Maxwell et al 1991) 5 UK Case reports Fetus A Referred at 22 weeks gestation Procedure performed at 28 weeks Fetus B Referred at 30 weeks gestation Procedure performed at 31 and 33 weeks Disclosure of interest: one of the authors is supported by the British Heart Foundation and another author by the British Heart Foundation and the Joseph Levy Foundation. Fetus A Aortic valve could not be crossed and three episodes of fetal bradycardia occurred during the procedure. The attempt was considered to have failed. The fetus died in the next 24 hours. Examination revealed a single puncture mark on the anterior chest wall. The aortic valve had been torn during the procedure. Fetus B Balloon ruptured and detached. Procedure was deemed to be unsuccessful. A second attempt was made 10 days later fetus experienced transient bradycardia. Baby was delivered spontaneously, weighing 2.98kg with Apgar scores of 7 and 9 at 1 and 5 minutes. A further procedure was performed in the newborn period neonate died at 28 days. Complications see efficacy section This is the first published report of fetal cardiac intervention for severe aortic stenosis. Background: 28 fetuses were identified with aortic stenosis. 12 mothers elected to continue pregnancy 2 fetuses died in utero and none of the 10 births survived. Balloon catheters that were used were a modification of those used in postnatal surgery. Lopes (1996) 3 Brazil Case report Fetus with critical aortic stenosis, diagnosed in utero at 27 weeks gestation. Authors note that since the endocardium at this stage was of normal appearance, it was hoped the procedure would prevent the development of endocardial fibroelastosis and irreversible damage to the left ventricle. Disclosure of interest: not stated While being withdrawn through the needle, the catheter scraped on the edge of the needle and the balloon was torn. Procedure was still considered a technical success. However follow-up showed no change in the left ventricle. An elective caesarean section was performed at 38 weeks. Baby weighed 2750g, with Apgar scores of 8 and 9 at 1 and 5 minutes. Infant underwent further procedures in the newborn period but died in the first day of life. Complications see efficacy section Included in later report 6 Case report highly selected patient. Authors note that selection of the patients is important in determining the success of this procedure. IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 8 of 19

9 Abbreviations used: AS aortic stenosis, LV left ventricle, HLHS hypoplastic left heart syndrome, PAIVS pulmonary atresia with intact ventricular septum Study Details Key efficacy and safety findings Key efficacy and safety findings Comments Allan et al (1995) 2 Italy/UK Case report Fetus was referred at 32 weeks gestation; procedure was performed at 33 weeks. Benson (2005) 7 Case series - 34 fetuses with gestational age weeks (mean 24 weeks) Outcome of the first 14 fetuses following successful intervention was compared to a control group of 4 fetuses whose parents declined the procedures and 6 fetuses with unsuccessful intervention. Selection criteria: fetuses with critical AS developing HLHS (further criteria according to intervention protocol but not mentioned in abstract). Bradycardia occurred during the procedure. Baby was delivered at 38 weeks weighing 2.9kg however the gradient across the aortic valve had not decreased. Further intervention was performed during the newborn period. The child was well at 4 years after the procedure. Aortic valve dilatation was successful in 27 cases and unsuccessful in 7 cases. Outcomes in the first 14 fetuses after successful aortic valve dilation demonstrated significant growth of the mitral valve, aortic valve and ascending aortic and a trend towards left ventricular growth compared to 10 control fetuses. 3/14 fetuses with successful intervention had adequate left ventricular function for a twoventricle heart as compared to none of the controls. 2/27 fetuses died in utero (one with severe hydrops and bradycardia prior to intervention) as did 2/10 fetuses in the control group. Complications see efficacy section Included in later report 6 Complications see efficacy section Abstract limited information. Unclear what is meant by success assume it is technical. Authors note that success rate improved with experience only one of the first four procedures was successful as compared to 26 of the last 30. IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 9 of 19

10 Abbreviations used: AS aortic stenosis, LV left ventricle, HLHS hypoplastic left heart syndrome, PAIVS pulmonary atresia with intact ventricular septum Study Details Key efficacy and safety findings Key efficacy and safety findings Comments Gardiner (2005) 8 Case series UK 6 fetuses (7 procedures) - 4 PAIVS - 3 critical aortic stenosis Mean age: weeks gestation Follow-up: maximum 5 years Disclosure of interest: not stated. Six procedures were technically successful. Three with hydrops (1 PAVIS 2 aortic stenosis) showed resolution and improved circulatory indices. All had good forward flow after procedure but restenosis occurred in 4 (2 PAIVS and 2 AS). All three fetuses with aortic stenosis presented with dilated left ventricles, endocardial fibroelastosis, restrictive oval foramen and abnormal pulmonary venous indices. Ventricular growth and improved function was observed with normalisation of pulmonary venous flow. One fetus with aortic stenosis had successful neonatal Ross-Konnon but had pulmonary hypertension managed using home nitric oxide at 3 years. The second fetus with aortic stenosis showed borderline left ventricular size at birth and underwent a Norwood procedure. The third AS fetus suffered cerebral haemorrhage and treatment was withdrawn. Complications see efficacy section Abstract limited information. Includes fetuses with either pulmonary atresia or aortic stenosis Limited information IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 10 of 19

11 Validity and generalisability of the studies There is limited published evidence on this procedure, and the studies that are published are small case series or case reports (total published n=38). The procedure is undertaken in severe and highly selected cases. Given that cases are rare, it is unlikely that there will be a substantial body of published evidence in the near future. Despite cases being highly selected, there is still substantial variation in terms of presenting disease. Selection criteria for the intervention are still being defined. There appears to be a learning curve associated with this procedure with some authors noting a high rate of technical failure in the first few fetuses treated 4,7 There is a lack of long-term data. Specialist advisors opinions Specialist advice was sought from consultants who have been nominated or ratified by their Specialist Society or Royal College. Dr H Gardiner, Mr D Howe, Professor Kilby, Mr Gerald Mason, Professor S Robson, Professor C Rodeck, Dr G Sharland, Professor PW Soothill, Dr O Stumper, Mr S Walkinshaw, Dr C Wren. The comparison would be to wait until after birth to perform surgical intervention. There is uncertainty around whether fetal cardiac procedures are beneficial or necessary. The main difficulty is being able to predict the fetal outcome prenatally (issues around whether HLHS can be prevented or does it progress from aortic stenosis). There is a lack of long term data. The procedure has a potentially large impact but on a very small number of individuals. The procedure is usually considered to be indicated in fetuses with severe aortic valve stenosis which is thought to progress to HLHS. The procedure should be performed in a specialised fetal medicine unit with high quality imaging, trained midwives and the ability to deliver the baby urgently if required. Learning curve is associated with the success of the procedure. Issues for consideration by IPAC There is a voluntary European database on fetal cardiac interventions held by two members of the Fetal Working Group of the Association of European Paediatric Cardiologists IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 11 of 19

12 References 1 Tworetzky W and Marshall AC. (2003) Balloon valvuloplasty for congenital heart disease in the fetus. Clinics in Perinatology.Vol.30(3)()(pp ), Allan LD, Maxwell DJ, Carminati M et al. (1995) Survival after fetal aortic balloon valvoplasty. Ultrasound in Obstetrics & Gynecology.5(2):90-1, 3 Lopes LM, Cha SC, Kajita LJ et al. (1996) Balloon dilatation of the aortic valve in the fetus. A case report. Fetal Diagnosis & Therapy.Vol.11(4)()(pp ), Tworetzky W, Wilkins-Haug L, Jennings RW et al. (2004) Balloon dilation of severe aortic stenosis in the fetus: Potential for prevention of hypoplastic left heart syndrome. Candidate selection, technique, and results of successful intervention. Circulation.Vol.110(15)()(pp ), 2004.Date of Publication: 12 OCT Maxwell D, Allan L, and Tynan MJ. (1991) Balloon dilatation of the aortic valve in the fetus: A report of two cases. British Heart Journal.Vol.65(5)()(pp ), Kohl T, Sharland G, Allan LD et al. (2000) World experience of percutaneous ultrasoundguided balloon valvuloplasty in human fetuses with severe aortic valve obstruction. American Journal of Cardiology.Vol.85(10)()(pp ), 2000.Date of Publication: 15 MAY Benson CB. (2005) Developments in the dilatation of cardiac valves. Ultrasound in Obstetrics & Gynecology.16(3):275-8, 26: Gardiner HM. (2005) Percutaneous fetal valvuloplasty: four years experience. Presentation AHA Dallas November 2005 IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 12 of 19

13 Appendix A: Additional papers on percutaneous fetal aortic balloon valvuloplasty No additional literature was identified at the time of the literature search. IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 13 of 19

14 Appendix B: Related NICE guidance for percutaneous fetal aortic balloon valvuloplasty Programme Interventional Procedures Recommendation IPG 078 Balloon valvuloplasty for aortic valve stenosis Recommendation: 1.1 Current evidence on the safety and efficacy of balloon valvuloplasty for aortic valve stenosis in adults and children appears adequate to support the use of this procedure provided that the normal arrangements are in place for consent, audit and clinical governance. 1.2 In adults, the procedure should only be used to treat patients who are unsuitable for surgery, as the efficacy is usually shortlived. 1.3 In infants and children, the procedure should be undertaken in specialist paediatric cardiology units. 1.4 The Department of Health runs the UK Central Cardiac Audit Database (UKCCAD) and clinicians are encouraged to enter all patients onto this database ( IPG067 Balloon dilatation of pulmonary valve stenosis Recommendation: 1.1 Current evidence on the safety and efficacy of balloon dilatation of pulmonary valve stenosis appears adequate to support the use of this procedure, provided that the normal arrangements are in place for consent, audit and clinical governance. 1.2 Balloon dilatation of pulmonary valve stenosis should only be performed in a specialist unit where paediatric cardiac surgery is available. 1.3 The Department of Health runs the UK Central Cardiac Audit Database (UKCCAD) and clinicians are encouraged to enter all patients onto this database ( Other Comments: This procedure has become established practice on the basis of clinical experience. There is very limited research evidence published. Most of the data relates to neonates and children, but the procedure can also be performed in adults. IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 14 of 19

15 IPG075 Balloon angioplasty of pulmonary vein stenosis in infancy Recommendation: 1.1 Current evidence on the safety and efficacy of balloon angioplasty of pulmonary vein stenosis in infants does not appear adequate for this procedure to be used without special arrangements for consent and for audit or research. The available evidence suggests that the procedure is not efficacious. However, there are no special concerns about the safety of the procedure, especially in the context of very ill infants for whom it is used. 1.2 Clinicians wishing to undertake balloon angioplasty of pulmonary vein stenosis in infants should take the following actions: Inform the clinical governance leads in their Trusts. Ensure that the parents of patients understand that the limited available evidence indicates a lack of efficacy. Parents should be given clear written information. Use of the Institute s Information for the Public is recommended. Audit and review clinical outcomes of all patients having balloon angioplasty of pulmonary vein stenosis in infancy. 1.3 This procedure should only be offered to gravely ill infants with a very poor prognosis and in the setting of a specialist paediatric cardiology unit. 1.4 The Department of Health runs the UK Central Cardiac Audit Database (UKCCAD) and clinicians are encouraged to enter all patients onto this database ( 1.5 Publication of safety and efficacy outcomes will be useful in reducing the current uncertainty. The Institute may review the procedure upon publication of further evidence. IPG076 Balloon dilatation with or without stenting for pulmonary artery or non-valvar right ventricular outflow tract obstruction in children IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 15 of 19

16 Recommendation: 1.1 Current evidence on the safety and efficacy of balloon dilatation with or without stenting for pulmonary artery or non-valvar right ventricular outflow tract obstruction in children appears adequate to support the use of this procedure, provided that the normal arrangements are in place for consent, audit and clinical governance. 1.2 The procedure should only be undertaken in specialist paediatric cardiology units. 1.3 The Department of Health runs the UK Central Cardiac Audit Database (UKCCAD) and clinicians are encouraged to enter all patients onto this database ( Other comments: Fewer data were available on the use of the technique for non-valvar right ventricular outflow tract obstruction than for pulmonary artery or branch pulmonary artery obstruction. IPG 077 Balloon dilation of systemic to pulmonary arterial shunts in children Recommendation: 1.1 Current evidence on the safety and efficacy of balloon dilatation of systemic to pulmonary arterial shunts in children appears adequate to support the use of this procedure, provided that the normal arrangements are in place for consent, audit and clinical governance. 1.2 The procedure should only be undertaken in specialist paediatric cardiology units. 1.3 The Department of Health runs the UK Central Cardiac Audit Database (UKCCAD) and clinicians are encouraged to enter all patients onto this database ( IPG 095 Radiofrequency valvotomy for pulmonary atresia Recommendation: 1.1 Current evidence on the safety and efficacy of radiofrequency valvotomy for pulmonary atresia with intact interventricular septum is limited due to the rarity of the condition, but appears adequate to support the use of the procedure for the treatment of seriously ill neonates, provided that normal arrangements are IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 16 of 19

17 in place for consent, audit and clinical governance. 1.2 Radiofrequency valvotomy for pulmonary atresia with intact interventricular septum should be performed in carefully selected patients in specialist centres with paediatric cardiac surgery facilities. 1.3 The Department of Health runs the UK Central Cardiac Audit Database (UKCCAD) and clinicians are encouraged to enter all patients onto this database ( Other comments: In making its recommendations, the Advisory Committee was influenced by the specialist advice that the procedure is established treatment for severely ill neonates who may otherwise die. Technology Appraisals Clinical Guidelines Public Health None relevant None relevant None relevant IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 17 of 19

18 Appendix C: Literature search for percutaneous fetal aortic balloon valvuloplasty Databases Version searched (if applicable) Date searched The Cochrane Library The Cochrane Library 2005, Issue 3 19/09/2005 CRD 21/09/2005 Embase 1980 to 2005 Week 37 19/09/2005 Medline 1966 to September Week /09/2005 Premedline September 15, /09/2005 CINAHL 1982 to September Week British Library Inside Conferences (limited to current year only) National Research Register 19/09/ /09/ /09/2005 Controlled Trials Registry 21/09/2005 The following search strategy was used to identify papers in Medline. A similar strategy was used to identify papers in other databases. Search strategy used in Medline 1. valvuloplast$.tw. 2. valvoplast$.tw. 3. valvotom$.tw. 4. exp balloon dilatation/ 5. (balloon adj3 (dilation or dilatation)).tw. 6. ptpv.tw. 7. or/ pulmonary valve/ 9. pulmonary atresia/ 10. pulmonary valve stenosis/ 11. pulmon$.tw. 12. heart defects, congenital/ 13. (congenital adj3 (cardiac or cardiovascular or heart$) adj3 (disease$ or defect$)).tw. 14. aortic valve/ 15. aortic valve stenosis/ 16. aortic$.tw. 17. or/ and fetus/ IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 18 of 19

19 20. fetal heart/ 21. fetal diseases/ 22. (fet$2 or foet$2).tw. 23. or/ and animal/ not human/ not 25 IP Overview: percutaneous fetal aortic balloon valvuloplasty Page 19 of 19