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
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Germ cell tumors
- Poor initial response
- Short initial response
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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
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Ewing family of tumors
- Metastatic disease at diagnosis
- First relapse or CR2
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Medulloblastoma
- First relapse or CR2
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Transplant Consultation Guidelines: Other Non-Malignant Diseases
Hemophagocytic lymphohistiocytosis (HLH)
- At diagnosis
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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)
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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)
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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
- Tefferi A. How I treat myelofibrosis. Blood. 2011; 117(13): 3494-3504. Access
- 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
- 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
- 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
- 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
- Hale GA, Arora M, Ahn KW. Allogeneic hematopoietic cell transplantation for neuroblastoma: The CIBMTR experience. Bone Marrow Transplant. 2013; 48(8): 1056-1064. Access
- 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
- 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
- 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
- Seo JJ. Hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis: recent advances and controversies. Blood Res. 2015; 50(3): 131-139. Access
- 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
- 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
- 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
- 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
- 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
- Asano Y. Systemic sclerosis. The Journal of Dermatology. 2018;45(2):128-138. doi:10.1111/1346-8138.14153. Access
- 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
- NMDP/Be The Match and ASTCT Recommended Timing for Transplant Consultation. Download (PDF)
- National Comprehensive Cancer Network. NCCN Guidelines. 2023. Access