Disease-Specific Indications and Outcomes

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Other and Autoimmune Diseases  

Chemotherapy is a common treatment for many patients with the diseases listed below. However, hematopoietic cell transplantation (HCT) may also be indicated. 

Malignant Diseases  

Myelofibrosis (MF)  

Allogeneic HCT may be the only curative treatment option for MF. Risks must be weighed along with the best conditioning regimen for the patient according to disease process and severity. MF is a rare myeloproliferative neoplasm primary myelofibrosis that occurs in older adults with a variable prognosis. [1-3] 

Germ cell tumors  

Ovarian cancer 

Ovarian cancer is a leading cause of death for women in the United States, and research is continuously evolving to show the clear risks and benefits of allogeneic HCT. [4] 

Testicular cancer 

Male germ cell tumors are one of the most common curable malignancies among young adult men. Autologous HCT in early relapse and tandem transplantation show outcomes have improved over time. [5] 

Neuroblastoma  

Autologous HCT has been well established as a treatment option for patients with neuroblastoma. However, allogeneic HCT may provide decreased relapse and prolonged survival, but more research is needed to narrow immunological parameters to reduce disease recurrence in patients with advanced disease. [6,7] 

Ewing family of tumors  

This aggressive sarcoma is optimized by chemotherapy or surgical radiation, but several clinical trials have expanded therapy combinations to improve survival. [8] 

Medulloblastoma  

Medulloblastoma is the most common malignant brain tumor in the pediatric population. Advanced studies of this complex cancer's biology have led to defined treatment regimens and clinical trials involving autologous HCT. [9]

Non-Malignant Diseases 

Hemophagocytic lymphohistiocytosis (HLH)  

Early donor search is essential for patients newly diagnosed with HLH. Allogeneic HCT is a treatment indicated for recurrent or progressive HLH that may improve outcomes over time, especially with ongoing efforts to optimize pre-transplant treatment. [10] 

Autoimmune Diseases  

Multiple Sclerosis [H3] 

Multiple sclerosis (MS) is a chronic, autoimmune, degenerative disease affecting nearly 1 million adults in the United States. [11] Most patients present with symptoms between 20 and 40 years of age but have varying rates of disability impairment. [12] 

There is currently no cure for MS. However, research has shown that autologous HCT is an efficient and safe treatment for active relapsing forms of MS to help prevent relapse, lesion-activity, and worsening disability while limiting unexpected adverse events. [12] In addition, patients with aggressive forms of MS may particularly benefit from transplant accompanied by high-dose immunotherapy. [13,14] Together, these treatments suppress the autoimmune inflammatory response and promote immune recovery. 

The American Society for Transplantation and Cellular Therapy (ASTCT) has recommended autologous transplant be considered for “standard of care, clinical evidence available” treatment of refractory-relapsing MS. [12] 

Systemic Sclerosis  

Systemic sclerosis (SS) or scleroderma is a rare autoimmune disease affecting approximately 50 per 100,000 people in the United States. [15] It is characterized by debilitating effects on the connective tissue and various internal organs, caused by genetic and environmental predispositions. [16] 

The use of autologous or allogeneic HCT is being considered for the treatment of SS. Preclinical and clinical studies suggest that high-dose immunotherapy alongside allogeneic HCT may improve outcomes. [17] 

Risks related to the treatment regimen, graft-versus-host disease, relapse and overall transplant eligibility must be weighed when choosing the best option and stem cell source for transplant. [17] 

HCT Consultation Timing Guidelines  

The National Marrow Donor Program® (NMDP)/Be The Match® and the American Society for Transplantation and Cellular Therapy (ASTCT) have jointly developed guidelines for transplant consultation and referral timing based on disease characteristics. [18] The National Comprehensive Cancer Network Clinical Practice Guidelines (NCCN Guidelines®) were consulted when developing these guidelines and are a valuable tool in determining risk stratification. [19] 

Our guidelines highlight disease categories that include patients at risk for disease progression and who should be referred for a consultation for autologous or allogeneic transplantation.[18] 

Transplant Consultation Guidelines: Other Malignant Diseases 

Myelofibrosis (MF) 

  • DIPSS Intermediate-2 (INT-2) and high-risk disease 
  • DIPSS Intermediate-1 (INT-1) with low platelet counts, refractory, red blood cell transfusion dependent, circulating blast cells > 2% complex cytogenetics 
  • Metastatic disease at diagnosis 
  • Progressive disease while on therapy or relapsed disease 

Download as a slide 

Germ cell tumors 

  • Poor initial response 
  • Short initial response 

Download as a slide 

Neuroblastoma 

  • INSS stage 2 or 3 at diagnosis-MYSN amplification (>4x above reference) - age>18 months at diagnosis - age 12-18 months with unfavorable characteristics 
  • Metastatic disease at diagnosis 
  • Progressive disease while on therapy or relapsed disease 

Download as a slide

Ewing family of tumors 

  • Metastatic disease at diagnosis 
  • First relapse or CR2 

Download as a slide 

Medulloblastoma 

  • First relapse or CR2 

Download as a slide 

Transplant Consultation Guidelines: Other Non-Malignant Diseases  

Hemophagocytic lymphohistiocytosis (HLH) 

  • At diagnosis 

Download as a slide 

Transplant Consultation Guidelines: Multiple Sclerosis  

  • After MS relapse, with ≥ 2 relapse episodes in past 3 years, while on disease modifying therapy 
  • Refer patient prior to progression of severe disability: patient must be able to walk 100 meters (with unilateral assistance: cane, crutch or brace) 

Download as a slide 

Transplant Consultation Guidelines: Systemic Sclerosis  

HCT consultation should take place: 

  • At the time of diagnosis or with diffuse disease 
  • With increasing skin tightness score (modified Rodnan skin score, [mRSS])   
  • Evidence of decrease ( < 80%) in % predicted pulmonary function tests: forced vital capacity (FVC) or/and diffusion capacity (DLCO) 

Download as a slide 

View complete HCT Consultation Timing Guidelines  

Clinical Trials Search and Support  

The NMDP/Be The Match offers the Be The Match® Jason Carter Clinical Trials Search and Support (CTSS) program, which can provide clinical trial navigation to your patients. The CTSS Program was created to help people with blood cancers or blood disorders and their families find and join clinical trials. 

For more information, visit Clinical Trials Search and Support.  

References 

  1. Tefferi A. How I treat myelofibrosis. Blood. 2011; 117(13): 3494-3504. Access 
  2. Kröger NM, et al. Indication and management of allogeneic stem cell transplantation in primary myelofibrosis: a consensus process by an EBMT/ELN international working group. Leukemia. 2015; 29(11): 2126-2133. Access 
  3. Deeg HJ, et al. Hematopoietic cell transplantation as curative therapy for patients with myelofibrosis: Long-term success in all age groups. Biol Blood Marrow Transplant. 2015; 21(11): 1883-1887. Access 
  4. Bay J-O, Cabrespine-Faugeras A, Tabrizi R, et al. Allogeneic hematopoietic stem cell transplantation in ovarian cancer-the EBMT experience. International journal of cancer. 2010;127(6):1446-1452. doi:10.1002/ijc.25149. https://onlinelibrary.wiley.com/doi/10.1002/ijc.25149  Access 
  5. Kilari D, D’Souza A, Fraser R, et al. Autologous Hematopoietic Stem Cell Transplantation for Male Germ Cell Tumors: Improved Outcomes Over 3 Decades. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2019;25(6):1099-1106. doi:10.1016/j.bbmt.2019.02.015. Access
  6. Hale GA, Arora M, Ahn KW. Allogeneic hematopoietic cell transplantation for neuroblastoma: The CIBMTR experience. Bone Marrow Transplant. 2013; 48(8): 1056-1064. Access 
  7. Pinto NR, Applebaum MA, Volchenboum SL, et al.  Advances in Risk Classification and Treatment Strategies for Neuroblastoma.  J Clin Oncol. 2015;33:3008-3017. Access 
  8. Gaspar N, Hawkins DS, Dirksen U, et al.  Ewing Sarcoma: Current Management and Future Approaches Through Collaboration.  J Clin Oncol. 2015;33:3036-3046. Access 
  9. Juraschka K, Taylor MD. Medulloblastoma in the age of molecular subgroups: a review. Journal of neurosurgery Pediatrics. 2019;24(4):353-363. doi:10.3171/2019.5.PEDS18381. Access 
  10. Seo JJ.  Hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis: recent advances and controversies.  Blood Res. 2015; 50(3): 131-139.  Access 
  11. National Multiple Sclerosis Society. Landmark Study Estimates Nearly 1 Million in the U.S. Have Multiple Sclerosis. February 15, 2019. Accessed June 9, 2022.  Access 
  12. Cohen JA, Baldassari LE, Atkins HL, et al. Autologous Hematopoietic Cell Transplantation for Treatment-Refractory Relapsing Multiple Sclerosis: Position Statement from the American Society for Blood and Marrow Transplantation. Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation. 2019;25(5):845-854. doi:10.1016/j.bbmt.2019.02.014. Access 
  13. Muraro PA, Pasquini M, Atkins HL, et al. Long-term Outcomes After Autologous Hematopoietic Stem Cell Transplantation for Multiple Sclerosis. JAMA neurology. 2017;74(4):459-469. doi:10.1001/jamaneurol.2016.5867. Access 
  14. Nash RA, Hutton GJ, Racke MK, et al. High-dose immunosuppressive therapy and autologous HCT for relapsing-remitting MS. Neurology. 2017;88(9):842-852. doi:10.1212/WNL.0000000000003660. Access 
  15. Zhong L, Pope M, Shen Y, Hernandez JJ, Wu L. Prevalence and incidence of systemic sclerosis: A systematic review and meta-analysis. Int J Rheum Dis. 2019;22(12):2096-2107. doi:10.1111/1756-185X.13716. Access 
  16. Asano Y. Systemic sclerosis. The Journal of Dermatology. 2018;45(2):128-138. doi:10.1111/1346-8138.14153. Access 
  17. Sullivan KM, Sarantopoulos S. Allogeneic HSCT for autoimmune disease: a shared decision. Nature reviews Rheumatology. 2019;15(12):701-702. doi:10.1038/s41584-019-0306-7. Access
  18. NMDP/Be The Match and ASTCT Recommended Timing for Transplant Consultation. Download (PDF) 
  19. National Comprehensive Cancer Network. NCCN Guidelines. 2023. Access