Heart and heart-lung transplantation for idiopathic restrictive cardiomyopathy in children

Abstract

Objective: To review the outcome of cardiac transplantation for restrictive cardiomyopathy (RCM) in children and to assess the ability of new strategies to modulate the effects of high pulmonary vascular resistance. Design: Retrospective case note analysis of all patients receiving a transplant for RCM. Patients: 18 children with RCM referred for transplantation assessment to Great Ormond Street Hospital, London. Results: Eight boys and 10 girls were referred for assessment. Median age at presentation was 5.0 (mean (SD) 6.1 (4.0)) years. Fourteen orthotopic and two heterotopic transplantations were performed and two patients were referred for heart-lung transplantation. Mean duration from diagnosis to transplantation was 3.3 (3.0) years. Three patients with haemodynamic decompensation before transplantation had increased morbidity in the postoperative period. No patients died while awaiting a transplant. Three patients died in the first year after transplantation, one within 30 days. Five patients received pretransplantation prostacyclin for a mean duration of 57 (18) days. Transpulmonary gradient was reduced in four of the patients. Mean transpulmonary gradient was 27 (9.8) mm Hg before and 17 (6.7) mm Hg after treatment with prostacyclin (p , 0.05). Conclusion: Most children with RCM require transplantation within four years of diagnosis. Referral for transplantation assessment should precede haemodynamic decompensation. Increase of pulmonary vascular resistance is a variable problem but can be modulated with pre-transplantation prostacyclin. With these strategies, orthotopic transplantation is possible in the majority of cases.

Pediatric cardiomyopathies: A review of literature on clinical status and meta-analysis of diagnosis and clinical management methods

“…Its most common indication if the ECM phenotype [81,83,85]. In addition to pharmacotherapy and heart transplantation, other heart failure therapies such as mechanical circulatory support and LC assist devices (LVAD) are common clinical management therapies assessed by 18% of the studies.…”

“…Reported long-term mortality rates range between 0% [87] and 13% [83] compared with 38% without heart transplantation [82]. The most frequent indication for heart transplantation is pediatric patients diagnosed with the RCM phenotype [81][82][83][85][86][87] …”

“…Two studies did not specify the type of pediatric cardiomyopathy [84,88]. The dominant clinical management methods were heart transplantation assessed by 44% of the studies [63,[81][82][83][85][86][87] followed by pharmacotherapy, which was similarly assessed by 44% of the studies [63,79,80,[89][90][91][92] [78,84,88]. The main clinical outcomes assessed in the studies was five-year survival rate and/or reported mortality rate but the period differed in individual studies.…”

“…Combined five-year survival rate was 69% and mortality rate of 12%. The most commonly studied phenotype of pediatric cardiomyopathy was DCM by 50% of the studies [63,79,85,86,[89][90][91][92] followed by RCM (37.5% of the studies) [81][82][83][85][86][87] and finally HCM (12.5% of the studies) [78,80]. Two studies did not specify the type of pediatric cardiomyopathy [84,88].…”

“…Sixteen (16) studies conducted between 1969 and 2013 meeting the inclusion criteria were included in this meta-analysis [63,[78][79][80][81][82][83][84][85][86][87][88][89][90][91][92]. The total pediatric patients in all the 16 studies was 1,021 with mean age 7.73 years and mean follow-up period of 6.8 months.…”