Left ventricular assist device as destination therapy in cardiac end-stage dystrophinopathies: Midterm results.
We report our experience with the use of a left ventricular assist device (LVAD) as destination therapy (DT) for the management of patients with cardiac end-stage dystrophinopathies.



From February 2011 to February 2016, 7 patients with dystrophinopathies and dilated cardiomyopathy (DCM) were treated with LVADs at our institution. Median age at surgery was 16.5 years (range, 14.2-23.4 years). All patients were preoperatively evaluated by a multidisciplinary team approach.



All patients survived to hospital discharge. The early postoperative course was characterized by abdominal bleeding (1 patient) and retropharyngeal bleeding (1 patient). Because of abdominal or retropharyngeal bleeding, both required postoperative heparin infusion discontinuation for 35 and 33 days, respectively. Among the late complications, 1 child developed osteolysis and infection at the pedestal site of the device, which required surgical displacement; 1 patient required gastrostomy as a result of poor feeding, and another had a cerebral stroke, which was treated with percutaneous thrombus aspiration. The other 2 patients did not show early or late complications. At a median follow-up time of 21.7 months (range, 3-45 months) there have been 3 deaths: 1 patient died of a lung infection after 45 months, 1 died of tracheal bleeding after 29 months, and 1 died of cerebral hemorrhage after 14 months.



Our experience indicates that the use of an LVAD as DT in patients with dystrophinopathies with end-stage DCM is feasible, suggesting that it may be suitable as a palliative therapy for the treatment of these patients with no other therapeutic options.
Department of Pediatric Cardiology and Cardiac Surgery, Bambino Gesù Children Hospital, IRCCS, Rome, Italy; Pneumology Unit - University Hospital Pediatric Department, Bambino Gesù Children Hospital, IRCCS, Rome, Italy; Cardiovascular Science, Cardio-Thoracic Surgery Unit, Catholic University, A. Gemelli Hospital, Rome, Italy. Electronic address: dr.gianluigiperri@gmail.com.
Gianluigi
G
Perri
Department of Pediatric Cardiology and Cardiac Surgery, Bambino Gesù Children Hospital, IRCCS, Rome, Italy.
Sergio
S
Filippelli
Department of Pediatric Cardiology and Cardiac Surgery, Bambino Gesù Children Hospital, IRCCS, Rome, Italy.
Rachele
R
Adorisio
Department of Pediatric Cardiology and Cardiac Surgery, Bambino Gesù Children Hospital, IRCCS, Rome, Italy.
Roberta
R
Iacobelli
Department of Pediatric Cardiology and Cardiac Surgery, Bambino Gesù Children Hospital, IRCCS, Rome, Italy.
Francesca
F
Iodice
Department of Pediatric Cardiology and Cardiac Surgery, Bambino Gesù Children Hospital, IRCCS, Rome, Italy.
Giuseppina
G
Testa
Pneumology Unit - University Hospital Pediatric Department, Bambino Gesù Children Hospital, IRCCS, Rome, Italy.
Maria Giovanna
MG
Paglietti
Cardiovascular Science, Cardio-Thoracic Surgery Unit, Catholic University, A. Gemelli Hospital, Rome, Italy.
Domenico
D
D'Amario
Cardiovascular Science, Cardio-Thoracic Surgery Unit, Catholic University, A. Gemelli Hospital, Rome, Italy.
Massimo
M
Massetti
Department of Pediatric Cardiology and Cardiac Surgery, Bambino Gesù Children Hospital, IRCCS, Rome, Italy.
Antonio
A
Amodeo
D000293:Adolescent; D006328:Cardiac Catheterization; D002311:Cardiomyopathy, Dilated / Q000175:diagnosis / Q000503:physiopathology / Q000601:surgery*; D004452:Echocardiography; D005260:Female; D005500:Follow-Up Studies; D006333:Heart Failure / Q000175:diagnosis / Q000503:physiopathology / Q000601:surgery*; D006352:Heart Ventricles / Q000000981:diagnostic imaging / Q000503:physiopathology*; D006353:Heart-Assist Devices; D006801:Humans; D019028:Magnetic Resonance Imaging, Cine; D008297:Male; D010166:Palliative Care; D012189:Retrospective Studies; D056346:Sternotomy / Q000379:methods*; D013318:Stroke Volume / Q000502:physiology*; D014057:Tomography, X-Ray Computed; D016896:Treatment Outcome; D016277:Ventricular Function, Left / Q000502:physiology; D055815:Young Adult
Duchenne muscolar dystrophy; left ventricular assist device