Dear IPTA Colleagues,

I hope this message finds you well and that you have had a fantastic summer/winter filled with moments of relaxation and rejuvenation.

I am thrilled to share some exciting updates with you regarding the progress and initiatives within IPTA. The newly assembled IPTA committees have been hard at work, brimming with enthusiasm and creativity to enhance the impact of our organization. The Ethics Committee, for instance, has conducted a comprehensive survey around non-adherence and is currently setting an agenda to prioritize essential new initiatives. This commitment to ethical considerations will undoubtedly strengthen IPTA's mission and operations.

Furthermore, I am delighted to inform you that planning for the program for next years’ Fellows and AHNP educational virtual meeting is in the works and making significant progress. This educational program is essential to foster continuous learning and development among our members. In line with this, the Education Committee has finalized a survey for Fellows to better understand your educational needs. Your valuable input will lay the foundation for our 2024 educational activities, ensuring they align perfectly with what you seek. Please take a look at the Education Committee update in this Newsletter to access this survey in English and Spanish. Planning from the AHNP side will start soon.

As we look ahead to the future, I am excited to share that the 2025 Congress Scientific Program Committee is already working diligently on organizing the IPTA congress in Berlin for 2025, led by Priya Verghese. We eagerly welcome any ideas that directly reach Priya from the IPTA membership. Your suggestions will help shape the congress's program, making it as relevant and beneficial to you as possible. Additionally, under the guidance of Anette Melk and Chesney Castleberry, we will be introducing a new mentoring program as part of the 2025 congress, which promises to be an invaluable opportunity for personal and professional growth.

To encourage collaboration and specialized discussions, IPTA will be implementing organ-specific communities that will be open to all members. These communities will not only contribute to the development of IPTA guidelines but also serve as forums for discussions on organ-specific themes that are or may become significant. I encourage each of you to actively engage with these self-organized groups, as your input will be instrumental in shaping IPTA's direction and impact.

As we move forward with these exciting developments, I extend my heartfelt gratitude to each member of IPTA. Your support, involvement, and dedication make all of this possible. Together, we will continue to advance pediatric transplant medicine and make a positive difference in the lives of countless individuals.

Should you have any suggestions, queries, or ideas, please don't hesitate to reach out. Your voice matters, and I am here to listen and act upon your input.

Thank you all for your unwavering commitment to IPTA. Together, we will achieve remarkable accomplishments.

Yours faithfully,

Lars Pape
IPTA President, 2023-2025

Update

Submitted by Dr. Gillian Mayersohn, on behalf of the IPTA Allied Health & Nursing Professionals (AHNP) Committee

New Committee Members
Welcome to Jenny Prüfe as the new co-chair of the AHNP. Congratulations to Ashley Graham on finishing her term as Co-Chair and moving into the Past Chair role. The committee also welcomes two new members – Alyssa Geis and Kat Broad. We thank all those members who have served on the committee and have transitioned off this year – we appreciate all of your time and support on the AHNP committee.

IPTA 2023 Congress Recap
AHNP conducted a pre-congress symposium focused on patient-specific ethical quandaries and family and socio-cultural quandaries. The pre-congress session featured engaging discussion from a variety of presenters. Throughout the remainder of the congress, there were poster and oral abstract sessions focused on a variety of allied health and nursing focused topics. These recordings are available for viewing by IPTA members in good standing on the IPTA homepage.

At the AHNP business meeting, allied health and nursing professionals along with other congress participants, engaged in a robust discussion of goals and upcoming project ideas for the AHNP committee. These ideas will be further discussed and developed in future AHNP meetings.

A big congratulations to Anna Gold PhD C.Psych on being the 2023 recipient of the Distinguished Allied Health and Nursing Professionals Award!

Upcoming Webinar
The ANHP committee will be working on partnering with Women in Transplantation on an upcoming webinar focusing on the role of women in transplant and academic medicine. Be on the lookout for more information to come soon!

Update on Transplantation for Methylmalonic Acidemia (MMA)

Submitted by Submitted by Dr. Riccardo Superina, on behalf of the IPTA Multi-Organ (MOTX) Committee

Methylmalonic Acidemia (MMA) is a rare inherited metabolic disorder in the family of organic acidemias. The disease is inherited in an autosomal recessive manner. The term isolated MMA refers to a group of inborn errors of metabolism associated with elevated MMA in the blood and urine that results from the failure to convert methylmalonyl-coenzyme A into succinyl-coenzyme A in the mitochondrial matrix. This is caused by a deficiency of methylmalonyl-CoA mutase (MMUT), resulting in accumulation of MMA.

Fig 1: The lower red X illustrates the step leading to accumulation of MMA from the lack of MMUT.1

The infantile non – B12 responsive form is the most common form. It is diagnosed in infancy, with newborn metabolic screens that assess of high levels of metabolic byproducts. Clinically, MMA is characterized by a variety of symptoms, including metabolic crises, neurocognitive delay and end-organ dysfunction. Furthermore, it is associated with various systemic sequelae, including optic, sensorineural hearing loss, cardiomyopathy, renal dysfunction, osteoporosis, and poor growth. To manage the accumulation of MMA, dietary restrictions are followed, with protein calorie restrictions, and frequent meals.

Solid organ transplantation, while not curative, is an important treatment in MMA. The three types of transplants that are done are kidney alone, liver alone or combined liver and kidney transplants. Organ transplantation practice varies with center. In young children in the absence of renal failure, isolated liver transplant has been described. Here, the potential benefits are improved metabolism of MMA prior to years of accumulation and cellular injury. On the other hand, older children with renal dysfunction have received combined liver and kidney transplants, as well as isolated kidney grafts.

Kidney transplantation alone in the setting of compromised renal function may result in long term improvement in renal function, but recurrence of nephropathy and metabolic instability are common. Kidney function may also deteriorate, and protein restriction and enteral nutritional strategies are still necessary. A significant number of recipients require retransplantation.2 In a French experience2, 50 % of kidney transplant recipients were considered for re-transplantation after a few years.

Early liver transplantation, before kidney deterioration occurs, may delay the need for kidney transplant for years and is done primarily for metabolic control in patients with preserved renal function. In an overview article of the current literature, Yal et al.3 reported that of 167 patients transplanted for MMA, 114 received a liver alone, while 32 received a combined liver-kidney transplant.

Cardiomyopathy associated with MMA may also be ameliorated following liver transplantation, although this is not a universal occurrence. Severe cardiomyopathy is a contraindication for transplantation.3

Combined liver and kidney transplants have been reserved primarily for those children with MMA and renal impairment3. Renal dysfunction in MMA may be due to intrinsic mitochondrial dysfunction from MMA accumulation and tubule-interstitial nephritis, simultaneous kidney transplantation is advised for improved renal function.

Contrary to the preponderance of liver transplants alone in the multicenter review, a single center experience4 reported that of 14 patients with MMA, 6 patients underwent a single liver transplant, while 8 had a combined liver-kidney transplant. All patients who received a combined transplant had compromised renal function and most were on hemodialysis.

Both liver and kidney grafts contain MMUT activity. While all recipients of either single kidney or liver transplants experience reduced levels of serum MMA, recipients with a liver graft have pronounced effect on MMA serum levels. However, patients who receive combined liver-kidney transplants, regardless of the preoperative renal function, experience more profound and longer lasting reductions of urinary MMA than either single kidney or liver transplants alone.2

While solid organ transplant alleviates symptoms due to metabolic crises, the build-up of MMA in the CNS persists. This can cause persistent neurologic symptoms and remains an ongoing challenge. The occurrence of metabolic crises after transplantation for MMA vary from center to center. Nakamura et al reported a high occurrence after liver transplant alone5, while others4 report no metabolic sequelae. Metabolic strokes have also been reported following liver transplants6 whereas this appears to be more uncommon as a complication after combined liver-kidney transplants.

While it is generally accepted that transplantation for MMA is not curative, and that metabolic perturbations in the central nervous system persist, the quality of life of patients with MMA improves after transplantation. A thoughtful approach needs to take place in determining the role of combined transplants, even in the absence of preexisting kidney disease. A multi-center collaboration may be necessary to provide the answer as to whether a combined liver and kidney transplant provides better and more long-lasting metabolic control and better long-term kidney function than liver transplant alone7 even ion patients with preserved renal function.

REFERENCES
  1. Forny P, Horster F, Ballhausen D, et al.
    Guidelines for the diagnosis and management of methylmalonic acidaemia and propionic acidaemia: First revision. J Inherit Metab Dis.2021;44(3):566-592
    https://pubmed.ncbi.nlm.nih.gov/33595124/
  2. Brassier A, Krug P, Lacaille F, et al.
    Long-term outcome of methylmalonic aciduria after kidney, liver, or combined liver-kidney transplantation: The French experience. J Inherit Metab Dis. 2020;43(2):234-243
    https://pubmed.ncbi.nlm.nih.gov/31525265/
  3. Yap S, Vara R, Morais A.
    Post-transplantation Outcomes in Patients with PA or MMA: A Review of the Literature. Adv Ther. 2020;37(5):1866-1896.
    https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7141097/
  4. Niemi AK, Kim IK, Krueger CE, et al.
    Treatment of methylmalonic acidemia by liver or combined liver-kidney transplantation J Pediatr. 2015;166(6):1455-1461 e1451
    https://pubmed.ncbi.nlm.nih.gov/25771389/
  5. Sakamoto R, Nakamura K, Kido J, et al.
    Improvement in the prognosis and development of patients with methylmalonic acidemia after living donor liver transplant Pediatr Transplant 2016;20(8):1081-1086. https://pubmed.ncbi.nlm.nih.gov/27670840/
  6. Morioka D, Kasahara M, Horikawa R, Yokoyama S, Fukuda A, Nakagawa A.
    Efficacy of living donor liver transplantation for patients with methylmalonic acidemia Am J Transplant. 2007;7(12):2782-2787.
    https://pubmed.ncbi.nlm.nih.gov/17908273/
  7. Sloan JL, Manoli I, Venditti CP
    Liver or combined liver-kidney transplantation for patients with isolated methylmalonic acidemia: who and when? J Pediatr 2015;166(6):1346-1350.
    https://pubmed.ncbi.nlm.nih.gov/25882873/

Update

Submitted by Dr. Pankaj Chandak and Dr. Manuel Rodriguez-Davalos on behalf of the IPTA Membership Communications Committee

The Membership and Communications committee is happy to share our focus on the diversity and the international representation from all continents following on from a fantastic IPTA meeting in Texas. It was so good to see friends and colleagues from all the world and to hear exciting science and innovation.

Creating a diverse and inclusive environment is a key component of our Association, our membership and communications committee is proud to have a balanced representation across pediatric specialties, gender and geographics, we would like to continue to make equity and inclusion a priority across IPTA and be a bridge in communications with transplant associations across the globe.

Pankaj Chandak (Co-Chair, Communications)
Manuel Rodriguez-Davalos (Co-Chair, Membership)
Vikas Dharnidharka (Council Liaison, Communications)
Deirdre Hahn (Council Liaison, Membership)
Devprakash Choudhary (Fellow)
Courtney Risley (AHNP Correspondent, Communications)
Grainne Walsh (AHNP Correspondent, Membership)
Ane Andres (Membership)
Carmen Capito (Membership)
Lyndsey Grover (Communications)
Lorraine Hamikwa (Communications)
Alison Maa (Communications)
Nima Memaran (Communications)
Scott McEwen (Membership)
Christine Hwang (Membership)
David Peng (Communications)
Sara Rasmussen (Membership)
Kathryn Shaw (Communications)
Sarbpreet Singh (Membership)
Nam Joon Yi (Communications)
Srinath Chinnakotla (Ex-Officio)

As part of this communications effort, IPTA worked with TTS and SPLIT on promoting the Pediatric Living Donors Webinar in Spanish through the Mexican chapter of AHPBA (AMHPB). Hosted by Dr. Daniel Zamora and featuring our Past President, Dr. Carlos Esquivel, and Committee Co-Chair, Dr. Manuel Rodriguez-Davalos, the webinar had a panel of 5 experts in the field. We believe it was a success, with 210 participants from more than six countries.

We will continue to share the projects of our committee members starting with the BBC Children In Need Harmonies of Hope Children’s Choir from our own Co-Chair, Pankaj. The Choir aims to develop an International Anthem for Children with Transplants and to collaborate with other similar choirs around the world.

Finally …We encourage all IPTA members to follow IPTA on Twitter and also use the handle to promote work done by committees and members around the world. We understand how busy our members can be and if you need information to be distributed, please contact our committee via Katie Tait, IPTA Section Manager at katie.tait@tts.org.

IPTA Fellows Survey

Submitted by Dr. Rupesh Raina, on behalf of the IPTA Education Committee

Calling all pediatric transplant Fellows! We are excited to invite you to be a crucial part of shaping the future of pediatric transplant fellowship education. As a dedicated member of the pediatric transplant community, your insights are invaluable to us.

At IPTA, we are committed to providing the highest quality of education to our fellows. To ensure that we meet your educational needs effectively, we have developed a comprehensive survey. This survey has been meticulously crafted to identify any gaps and areas of improvement in the existing fellowship education framework.

Your participation in this survey will play a pivotal role in shaping the future of pediatric transplant fellowship programs. Your responses to the 29 thoughtfully designed questions will contribute to refining the quality of education and crafting a holistic curriculum for aspiring pediatric transplant professionals like yourself.

The survey is conveniently available in both English and Spanish to cater to your preference. To get started, please click on the survey link provided below:

English Version

Spanish Version

We want to assure you that your responses will be treated with the utmost confidentiality. Your candid feedback will drive positive changes that benefit not only your educational journey but also the broader pediatric transplant community. Your time and contributions are highly appreciated, and we extend our heartfelt gratitude to you for taking part in this endeavour.

Thank you for your ongoing dedication to the field of pediatric transplantation.

Join IPTA Today!

Spread the word! If you know someone who you feel should become an IPTA member, please share this link https://tts.org/ipta-members-area/ipta-join or ask them to email katie.tait@tts.org

Let’s keep up this momentum and grow the Society to 1000+ members!

SAVE THE DATE
IPTA 2025!


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