Part 5: Allogeneic HSCT in refractory SJIA with lung disease; recent cases from centers in North America & Europe

Alexei A Grom; Scott W Canna; Rolla F Abu-Arja; Rashmi Sinha; Luciana Peixoto; Elvira Cannizzaro; Shanmuganathan Chandrakasan; Kyla Driest; Rebecca Marsh; Bénédicte Neven; Karen Onel; Sampath Prahalad; Susan Prockop; Pierre Quartier; Johannes Roth; Grant Schulert; Juliana M F Silva; Donna Wall; Ulrike Zeilhofer

doi:10.1186/s12969-023-00868-x

Part 5: Allogeneic HSCT in refractory SJIA with lung disease; recent cases from centers in North America & Europe

Pediatr Rheumatol Online J. 2024 Jan 5;21(Suppl 1):86. doi: 10.1186/s12969-023-00868-x.

Authors

Alexei A Grom¹, Scott W Canna^#², Rolla F Abu-Arja^#³, Rashmi Sinha^#⁴, Luciana Peixoto^#⁴, Elvira Cannizzaro^#⁵, Shanmuganathan Chandrakasan^{6

7}, Kyla Driest⁸, Rebecca Marsh⁹, Bénédicte Neven^{10

11}, Karen Onel¹², Sampath Prahalad⁶, Susan Prockop¹³, Pierre Quartier^{10

11}, Johannes Roth¹⁴, Grant Schulert¹⁵, Juliana M F Silva¹⁶, Donna Wall¹⁷, Ulrike Zeilhofer¹⁸

Affiliations

¹ Division of Rheumatology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH, USA. [email protected].
² Rheumatology & Immune Dysregulation, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
³ Department of BMT, Nationwide Children's Hospital, Columbus, OH, USA.
⁴ Systemic JIA Foundation, Cincinnati, OH, USA.
⁵ Department of Rheumatology, University Children's Hospital, Zurich, Switzerland.
⁶ Department of Pediatrics, School of Medicine, Emory University, Atlanta, GA, USA.
⁷ Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University, Atlanta, GA, USA.
⁸ Department of Rheumatology, Nationwide Children's Hospital, Columbus, OH, USA.
⁹ Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
¹⁰ Pediatric Hematology-Immunology and Rheumatology Department, Necker-Enfants- Malades University Hospital, Paris, France.
¹¹ Université Paris-Cité, Paris, France.
¹² Department of Rheumatology, HSS, New York City, NY, USA.
¹³ Dana Farber/Boston Childrens Hospital Center for Cancer and Blood Disorders, Boston, MA, USA.
¹⁴ Kantonsspital Luzern, University of Luzern, Luzern, Switzerland.
¹⁵ Division of Rheumatology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH, USA.
¹⁶ Department of BMT, Great Ormond Street Hospital for Children, London, UK.
¹⁷ 16. Blood and Marrow Transplant/Cellular Therapy, Division of Haematology/Oncology, Hospital for Sick Children Toronto, Toronto, Canada.
¹⁸ Department of BMT, University Children's Hospital, Zurich, Switzerland.

^# Contributed equally.

Abstract

It has been increasingly recognized that there is a subset of patients with refractory systemic JIA, who have failed all available medications and may benefit from HSCT. The increasing experience with HSCT in SJIA, suggests that despite the complicated post-HSCT course, short-term, the transplanted patients either achieved SJIA remission or reduced burden of disease. Longer follow-up, however, is needed to better define the long-term outcomes. The discussion at the NextGen 2022 conference was focused on the optimal timing for the procedure, the need for a good control of inflammatory SJIA activity prior to HSCT, and the role of the reduced intensity conditioning regimens as there was a remote concern that such regimens might increase the risk of SJIA relapse after the transplantation. There was unanimous agreement about the importance of long-term registries to address these questions.

Keywords: Allogeneic HSCT; HLA DRB1*15 alleles; MAS; Refractory SJIA; SJIA-LD.

MeSH terms

Europe
Hematopoietic Stem Cell Transplantation* / adverse effects
Humans
Lung Diseases* / therapy
North America
Registries