{"id":59413,"date":"2022-05-11T12:17:42","date_gmt":"2022-05-11T16:17:42","guid":{"rendered":"https:\/\/rarediseases.org\/nord-hosts-the-largest-gathering-of-a-rare-post-transplant-cancer-in-history\/"},"modified":"2022-05-11T12:17:42","modified_gmt":"2022-05-11T16:17:42","slug":"nord-hosts-the-largest-gathering-of-a-rare-post-transplant-cancer-in-history","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/nord-hosts-the-largest-gathering-of-a-rare-post-transplant-cancer-in-history\/","title":{"rendered":"NORD Hosts the Largest Gathering of a Rare, Post-Transplant Cancer in History"},"content":{"rendered":"

Post-Transplant Lymphoproliferative Disorder (PTLD) is a rare cancer related to organ and stem cell transplants, as well as Epstein bar disease. There is no nonprofit organization dedicated to this community and the last known gathering involved less than a dozen patients and caregivers, pre-pandemic. When NORD learned of interest in this disease to share patient and caregiver stories with the US Food and Drug Administration (FDA), we immediately started working with the patients and caregivers in related transplant communities with known advocates impacted by PTLD.<\/span>\u00a0<\/span><\/p>\n

When evaluating potential new treatments and reviewing clinical trials, the perspectives of patients and caregivers help government regulators (like FDA) and drug developers understand what matters most to those directly impacted by these drugs.<\/span> To share those community perspectives, NORD is proud to organize and host externally led Patient-Focused Drug Development (EL-PFDD) meetings, innovative events that provide an opportunity for families and patients to share critical information about the impact of a rare disease on their daily lives and provide valuable insight for FDA and other key stakeholders, including researchers, medical product developers and health care providers.<\/span>\u00a0<\/span><\/p>\n

On May 4, NORD hosted one of these critically important gatherings with FDA personnel, featuring the Post-Transplant Lymphoproliferative Disorder (PTLD) community. For the safety of the community, it was a fully virtual meeting, and featured live, engaging, interactive components, including polling, patient remarks, and engagement with federal decisionmakers and researchers. The four\u2013hour EL-PFDD meeting was host to over 100 attendees and featured honest testimonials from parents, family members, caregivers, advocates, and community members who shared the burdens and symptoms of PTLD and hope for more robust treatment and cures in the future. <\/span>\u00a0<\/span><\/p>\n

The goals of the\u00a0meeting were to tell stories and provide a robust understanding of how individuals with PTLD view their quality of life, which\u00a0aspects of the disease are most challenging for them, and what actions they currently take to treat this disease.\u00a0Caregivers and patients\u00a0shared\u00a0in-depth\u00a0stories\u00a0of how the disease\u00a0manifested in their lives and wreaked havoc on their livelihoods, health, relationships, and more. <\/span>\u00a0<\/span><\/p>\n

Swapna Kakani, who was diagnosed with PTLD after a small intestine transplant in 2014, kicked off the meeting with a powerful message about the stories shared in the meeting: \u201c<\/span>Our vulnerability in our words is to ask you to have compassion and meet us where we are today to have health AND life alongside a PTLD diagnosis. Both are not mutually exclusive.\u201d This set the tone for a productive and compelling conversation throughout the rest of the day.<\/span>\u00a0<\/span><\/p>\n

The audience\u00a0heard directly from patients who recounted their unpredictable daily struggles with the disease\u2019s widespread symptoms and what comes with such burdens,\u00a0including\u00a0from Ola Ojewumi, and the added challenges she faced from an inclusion standpoint. \u201cDuring cancer treatment, my clinicians spoke with me about treatment outcomes for African American patients or provide resources to learn more about the drug. I was hesitant to ask about race and ethnicity because discussions about race is still seen as taboo in society. I was afraid my concerns would be dismissed or seen as insignificant. Representation matters when it comes to clinical trials for cancer treatment.\u201d<\/span>\u00a0<\/span><\/p>\n

Parent and caregiver Greg Tufaro\u00a0spoke about the devastating\u00a0and consuming\u00a0impact of the disease on his late daughter, Marisa.\u00a0\u201c<\/span>My wife, Cyndi, and I are eternally grateful for the time God blessed us with Marisa. But we struggle daily to live with the tragic irony that the transplant performed to save her life inevitably claimed it, along with the heart of a donor whose family is also grieving\u2026 Marisa spent 161 of the last 214 days of her life as a patient at a nationally renowned children\u2019s hospital, where dozens of doctors and nurses, profoundly impacted by her remarkable courage and will to survive, were reduced to tears upon her untimely passing.\u201d<\/span>\u00a0<\/span><\/p>\n

Even for those such as Marianna DeLeon, who does not currently have signs of PTLD, there is still concern about what the future holds: \u201c<\/span>I am beyond happy to have had the amazing and unexpected gift of going into remission, but I will always bear the scars of not only my PTLD, but of the harsh, traumatic treatments I needed to survive it.\u201d<\/span>\u00a0<\/span><\/p>\n

However, this meeting was not just designed to share stories, but to make progress and build on lessons learned. The second half of the meeting focused on\u00a0treatments and facilitated audience discussion to urge strategic planning for next steps. The results of this meeting will be shared publicly in a \u201cVoice of the Patient\u201d report to inform the development of potential treatments that can improve the lives of patients living with PTLD and those who are diagnosed in the future.\u00a0 <\/span>\u00a0<\/span><\/p>\n

NORD would like to commend and recognize the great courage of all the panelists and speakers, and we are very grateful for their openness to share life experiences and needs. To view all the heartfelt stories and the entire meeting, please visit rarediseases.org\/externally-led-post-transplant-lymphoproliferative-disorder-patient-focused-drug-meeting<\/a>.<\/span><\/p>\n

NORD is thankful to have partners in the rare disease field interested in hearing patients\u2019 and caregivers\u2019 perspectives, who can help understand hopes for future treatments, treatment side effects patients are willing to tolerate, the medical risks they are willing to take in clinical trials, and their interests and challenges in participating in clinical trials. As Rebekah Palmer, a PTLD patient said in the meeting\u2019s closing remarks, \u201c<\/span>It is my greatest hope that these stories can help inform and improve the care received and quality of life for future generations living with PTLD.\u201d <\/span>\u00a0<\/span><\/p>\n

NORD applied to the FDA and was granted approval to host an EL-PFDD meeting focused on pyruvate kinase deficiency (PKD) in 2019, Krabbe disease in late 2020, PTLD last week, and is planning to host a PFDD in late 2022 for another rare disease, <\/span>Galactosemia,<\/span> in conjunction with the Galactosemia Foundation<\/span>. PFDDs are <\/span>one of the most important <\/span>way<\/span>s<\/span> the voice of rare disease patients and caregivers can be heard and utilized in decision making. Each year, NORD allocates resources and provides additional guidance and leadership to help a few groups <\/span>and disease communities with need in <\/span>host<\/span>ing<\/span> PFDDs and bring patient experiences directly to decisionmakers.<\/span><\/span>\u00a0<\/span><\/em><\/p>\n","protected":false},"excerpt":{"rendered":"

Post-Transplant Lymphoproliferative Disorder (PTLD) is a rare cancer related to organ and stem cell transplants, as well as Epstein bar disease. There is no nonprofit organization dedicated to this community … <\/p>\n

Continue reading “NORD Hosts the Largest Gathering of a Rare, Post-Transplant Cancer in History”<\/span><\/a><\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_relevanssi_hide_post":"","_relevanssi_hide_content":"","_relevanssi_pin_for_all":"","_relevanssi_pin_keywords":"","_relevanssi_unpin_keywords":"","_relevanssi_related_keywords":"","_relevanssi_related_include_ids":"","_relevanssi_related_exclude_ids":"","_relevanssi_related_no_append":"","_relevanssi_related_not_related":"","_relevanssi_related_posts":"","_relevanssi_noindex_reason":"","_price":"","_stock":"","_tribe_ticket_header":"","_tribe_default_ticket_provider":"","_tribe_ticket_capacity":"","_ticket_start_date":"","_ticket_end_date":"","_tribe_ticket_show_description":"","_tribe_ticket_show_not_going":false,"_tribe_ticket_use_global_stock":"","_tribe_ticket_global_stock_level":"","_global_stock_mode":"","_global_stock_cap":"","_tribe_rsvp_for_event":"","_tribe_ticket_going_count":"","_tribe_ticket_not_going_count":"","_tribe_tickets_list":"[]","_tribe_ticket_has_attendee_info_fields":false,"footnotes":""},"categories":[192,505],"tags":[203,270,2306,2998,244,2749,2304,2997,1812,383,2032],"class_list":["post-59413","post","type-post","status-publish","format-standard","hentry","category-patients-members","category-patient-stories","tag-cancer","tag-drug-development","tag-el-pfdd","tag-externally-led-patient-focused-drug-development","tag-fda","tag-partnership","tag-pfdd","tag-post-transplant-lymphoproliferative-disorder","tag-ptld","tag-research","tag-us-food-and-drug-administration"],"acf":[],"_links":{"self":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/59413","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/comments?post=59413"}],"version-history":[{"count":0,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/posts\/59413\/revisions"}],"wp:attachment":[{"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/media?parent=59413"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/categories?post=59413"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/rarediseases.org\/es\/wp-json\/wp\/v2\/tags?post=59413"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}