Case Study: Pediatric Dermatomyositis Associated with Acute Myeloid Leukemia - Successful Long-Term Remission Following Hematopoietic Stem Cell Transplantation - Report - MDSpire

Case Study: Pediatric Dermatomyositis Associated with Acute Myeloid Leukemia - Successful Long-Term Remission Following Hematopoietic Stem Cell Transplantation

  • By

  • Pan Fu

  • Kai Chen

  • Qing Liu

  • Bing Zou

  • Zhen Wang

  • Dan Wang

  • Hui Jiang

  • Na Zhang

  • Jingbo Shao

  • April 23, 2026

  • 0 min

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Clinical Report: Pediatric Dermatomyositis Associated with Acute Myeloid Leukemia

Overview

This case study presents a rare instance of juvenile dermatomyositis (JDM) progressing to acute myeloid leukemia (AML) in a pediatric patient, marking a significant finding in the literature. Following allogeneic hematopoietic stem cell transplantation (HSCT), both conditions achieved sustained remission at one-year follow-up.

Background

Juvenile dermatomyositis is a rare autoimmune disorder that can occasionally be associated with malignancies, including hematological cancers. The progression of JDM to AML in children is exceptionally rare, making this case significant for understanding potential treatment pathways. The successful use of HSCT in this context may provide insights into managing similar cases in the future.

Data Highlights

No numerical data or trial data presented in the article.

Key Findings

  • The patient was diagnosed with JDM at age 6, characterized by Gottron’s papules and muscle weakness.
  • Despite multiple immunosuppressive therapies, the patient experienced a relapsing-remitting course over 9 years.
  • At age 15, the patient was diagnosed with AML after presenting with knee arthralgia and fever.
  • Following induction chemotherapy and HSCT, both JDM and AML achieved sustained remission at 1-year follow-up.
  • This case suggests HSCT may be a viable treatment for refractory autoimmune-hematologic malignancy overlap syndromes.

Clinical Implications

This case highlights the importance of monitoring for hematological malignancies in pediatric patients with JDM, particularly when symptoms worsen. HSCT may offer a promising treatment option for patients with concurrent autoimmune diseases and hematological malignancies, warranting further investigation.

Conclusion

The successful long-term remission of both JDM and AML following HSCT in this patient underscores the potential of this treatment approach in complex cases. Further studies are needed to validate these findings and explore the broader implications for similar patients.

References

  1. Bone Marrow Transplantation, 2017 -- Initial Outcomes of Hematopoietic Stem Cell Transplantation for Shwachman-Diamond Syndrome Utilizing HLA-Matched Sibling Donors Identified via Preimplantation HLA Typing
  2. Blood Cancer Journal, 2023 -- Evaluation of the Safety and Effectiveness of CD22 and CD19 CAR-T Cell Therapy as a Bridging Strategy to Auto-Hematopoietic Stem Cell Transplantation for Adolescents and Young Adults with B-Cell Acute Lymphoblastic Leukemia
  3. Blood Cancer Journal, 2016 -- Pretransplant NPM1 MRD levels predict outcome after allogeneic hematopoietic stem cell transplantation in patients with acute myeloid leukemia
  4. Bone Marrow Transplantation, 2025 -- Indications for haematopoietic cell transplantation and CAR-T for haematological diseases, solid tumours and immune disorders: 2025 EBMT practice recommendations
  5. PubMed, 2025 -- Treating juvenile dermatomyositis to target: Paediatric Rheumatology European Society/Childhood Arthritis and Rheumatology Research Alliance-endorsed recommendations from an international task force
  6. BMC Cancer, 2025 -- Prognostic significance of cytogenetic and molecular features in pediatric acute myeloid leukemia: a meta-analysis
  7. Bone Marrow Transplantation — The 50th Annual Conference of the European Society for Blood and Marrow Transplantation: Physician Poster Presentations (P001-P755)
  8. Treating juvenile dermatomyositis to target: Paediatric Rheumatology European Society/Childhood Arthritis and Rheumatology Research Alliance-endorsed recommendations from an international task force - PubMed
  9. Prognostic significance of cytogenetic and molecular features in pediatric acute myeloid leukemia: a meta-analysis | BMC Cancer | Full Text
  10. Indications for haematopoietic cell transplantation and CAR-T for haematological diseases, solid tumours and immune disorders: 2025 EBMT practice recommendations | Bone Marrow Transplantation

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