There have been some important changes to the delamanid compassionate use protocol. Delamanid is available for compassionate use and can be obtained from the company at no charge on a patient-by-patient basis.
The company has made some changes to their access and eligibility criteria and these are summarized below. Perhaps most exciting is that the pediatric formulation of delamanid (a 25mg dispersible tablet) will also be available via compassionate use. This formulation is not yet on the market but is an important tool for younger children with RR-TB. We are thrilled the company has now made this formulation available for the children who need it.
Please find additional information below.
Updates to Delamanid Compassionate Use Protocol
- Male and female patients ≥ 3 years of age are eligible for CU DLM enrolment
- Prior delamanid use is no longer an exclusion criterion
DLM treatment duration
- The current protocol 242-302-00014 allows treatment extension of CU DLM on a case by case basis. Re-application for a treatment extension with delamanid can be made in exceptional cases, in order to minimize the probability of a resistance development to anti-TB treatment and/or enhance the likelihood of a favourable treatment outcome in difficult to treat cases.
- IMP of the pediatric formulation (dispersible tablets of 25 mg) is undergoing final packaging and testing and shall become available for patients enrolled in the updated CU protocol 242-302-00014 shortly.
PV reporting timelines
- The updated CU protocol 242-302-00014 requires reporting of all safety information within 24 hours (not just for SAE as in the previous CU protocol 242-12-401 but all AEs + safety information)
The Paediatric Drug-Resistant TB Donation Initiative is led and funded by the Stop TB Partnership’s Global Drug Facility in partnership and with financial support from USAID.
In June 2018, the South African National Department of Health announced that bedaquiline will now be used to replace the injectable agent in routine treatment of rifampin-resistant and multidrug resistant tuberculosis (RR/MDR-TB) in patients over 12 years of age. We congratulate the leadership of the South African National TB program for taking this important step.
We note with some concern that children under the age of 12 years may be precluded from accessing the benefits of an all-oral RR/MDR-TB regimen. As such, we call for policies to support all-oral regimens for patients under 12 years of age in South Africa and globally. In partnership with the Desmond Tutu Tuberculosis Centre (DTTC) and Treatment Action Group (TAG), Sentinel Project has developed the following recommendations for injectable-free regimens for children under the age of 12 years.
Please download these recommendations here: Recommendations for Injectible-Free Regimens in Children with Rifampacin Resistant TB.
The Sentinel Project resource, “How to Care for People Exposed to Drug-Resistant Tuberculosis: A Practical Guide” is now available.
This Guide builds on World Health Organization (WHO) recommendations for the management of individuals who have been exposed to TB and DR-TB, but focuses more on the practical implementation of interventions that should take place in the post-exposure setting. While the WHO policies stress that investigating household contacts of TB patients must be done on an “urgent” basis for all contacts of a patient with DR-TB, there is limited information available on what these evaluations and interventions should include. Furthermore, many of the recommendations and tools that do exist only focus on medical issues, omitting other pressing psychosocial needs that must be routinely assessed as well. DR-TB affects not only individuals but their households as well. Even when only one person has become sick, the whole household requires some intervention to reduce discrimination, improve patient support, and avoid unnecessary morbidity and mortality. This Guide aims to provide a way of thinking about the challenges around those exposed to DR-TB. It also aims to provide guidance on DR-TB prevention and management strategies needed to address the enormous health threat presented by DR-TB, as well as the tools necessary to carry them out.
For additional translations of the Field Guide, please send a note to Sentinel_Project@hms.harvard.edu.
On November 21, 2017, the Sentinel Project co-sponsored a webinar with DR-TB STAT, entitled “MDR-TB Treatment with Bedaquiline in Children and Adolescents: Global Recommendations and Program Experience in Belarus.” Dr. Alena Skrahina, MD, PhD, DSc, presented interim results of bedaquiline use in the treatment of children and adolescents with multi-drug resistant tuberculosis in Belarus. Dr. Skrahina discussed the acceptability of bedaquiline-containing regimens, culture conversion results, and the occurrence of adverse events. Dr. Skrahina was joined by Dr. Jennifer Furin as commentator and moderator.
On October 26, 2017, the Sentinel Project sponsored a presentation by Dr. Anthony Garcia-Prats and Dr. Anja Reuter entitled “Recent Developments in the Field of Pediatric MDR-TB.” Dr. Garcia-Prats reviewed emerging pharmacokinetic data on repurposed and novel second-line antituberculosis drugs in children, with a focus on implications for pediatric dosing. He reviewed ongoing pediatric trials and discuss expected data from these trials. Dr. Reuter discussed early experience with new TB drugs in early adolescents and children. She presented clinical cases of adolescents on treatment with new DR-TB drugs in a primary health care setting in Khayelitsha, South Africa. Dr. Garcia-Prats and Dr. Reuter were joined by Dr. Jennifer Furin as commentator and moderator. Slides from the webinar are available here.
On April 7, 2017 the Sentinel Project co-sponsored a webinar with DR-TB STAT, entitled “The Time Has Come: The Case for Injectable-Sparing Treatment Regimens for Children with MDR-TB.” Dr. Ethel Weld reviewed evidence and experience that supports the treatment of children with MDR-TB disease using regimens that do not require an injectable agent. Dr. Weld was joined by Dr. Kelly Dooley as commentator and Dr. Jennifer Furin as moderator. Slides from the webinar with Dr. Weld are available here, and the corresponding article published on March 23, 2017 in the Lancet Respiratory Medicine is available here.
On March 13, 2017, the World Health Organization issued a meeting report that included evidence on the use of bedaquiline in 537 individuals treated for MDR-TB disease, including adolescents. In light of this new data, a group of clinical experts from the Sentinel Project developed a brief update on bedaquiline, with recommendations to clinical providers and national TB programs. Please see the summary and recommendations here.